| Symbol | RARE |
|---|---|
| Name | ULTRAGENYX PHARMACEUTICAL INC. |
| Sector | HEALTH CARE |
| Region | North America |
| Industry | Biotechnology: Pharmaceutical Preparations |
| Address | 60 LEVERONI COURT, NOVATO, California, 94949, United States |
| Telephone | +1 415 - 483-8800 |
| Fax | — |
| — | |
| Website | https://www.ultragenyx.com |
| Incorporation | UNDEFINED |
| Incorporated On | — |
| Employees | — |
| Fiscal Year | — |
| Public Since | — |
| Exchanges | NASDAQ |
| Auditor | — |
| Audit Status | NOT PROVIDED |
| Reporting Status | — |
| CIK | 0001515673 |
| Description | Ultragenyx Pharmaceutical Inc is a US-based biopharmaceutical company. It identifies, acquires, develops, and commercialize novel products for the treatment of serious rare and ultra-rare diseases, with the focus on serious, debilitating genetic diseases. The companys medicine portfolio includes Crysvita and Mepsevii. Crysvita is indicated for the treatment of X-linked hypophosphatemia (XLH) in adult and pediatric patients 1 year of age and older. Additional info from NASDAQ: |
2026-07-08 13:08
Ultragenyx Releases 2025 Impact Report Emphasizing Commitment to Rare Disease Patients, Innovation, and Global Impact
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2026-07-06 19:37
Horn Howard 🔴 sold 4.7K shares of Ultragenyx Pharmaceutical Inc. (RARE) at $33.13 Transaction Date: Jul 01, 2026 | Filing ID: 296128
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2026-06-15 22:50
Sanders Corazon (Corsee) D. 🔴 sold 2.0K shares of Ultragenyx Pharmaceutical Inc. (RARE) at $25.05 Transaction Date: Jun 15, 2026 | Filing ID: 000007
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2026-06-15 22:46
Parschauer Karah Herdman 🔴 sold 1.9K shares of Ultragenyx Pharmaceutical Inc. (RARE) at $24.62 Transaction Date: Jun 15, 2026 | Filing ID: 000002
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2026-06-15 20:50
📋 KARAH PARSCHAUER (Officer) plans to sell 4K shares of ULTRAGENYX PHARMACEUTICAL INC. (at $24.35 each, total $93K) Filed: Jun 15, 2026 | ID: 006115
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2026-06-15 20:12
📋 CORAZON D. SANDERS (Director) plans to sell 2K shares of ULTRAGENYX PHARMACEUTICAL INC. (at $25.05 each, total $50K) Filed: Jun 15, 2026 | ID: 006073
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2026-06-12 14:40
New Form S-8 - Ultragenyx Pharmaceutical Inc. <b>Filed:</b> 2026-06-12 <b>AccNo:</b> 0001193125-26-269040 <b>Size:</b> 485 KB
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2026-06-04 20:36
Kuehn Law Encourages Investors of Ultragenyx Pharmaceutical Inc. to Contact Law Firm
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2026-06-03 20:30
Ultragenyx to Participate at Goldman Sachs 47th Annual Global Healthcare Conference
Read more| Trial ID | Title | Phase | Indication | Status | Start Date | Completion Date | Source |
|---|---|---|---|---|---|---|---|
| NCT03775174 | Expanded Access to Mepsevii | — | MPS VII | Available | — | — | ClinicalTrials.gov |
| NCT03773770 | Expanded Access to Triheptanoin | — | Long Chain Fatty Acid Oxidation Disorders | Available | — | — | ClinicalTrials.gov |
| NCT02968953 | Treatment With UX007 for a Single Patient With GLUT1 Deficiency Syndrome | — | Glucose Transporter 1 Deficiency Syndrome | No_Longer_Available | — | — | ClinicalTrials.gov |
| NCT02097251 | An Open-Label Treatment Protocol With UX003 rhGUS Enzyme Replacement Therapy fo… | — | Mucopolysaccharidosis Type 7 | No_Longer_Available | — | — | ClinicalTrials.gov |
| NCT01461304 | Compassionate Use of Triheptanoin (C7) for Inherited Disorders of Energy Metabo… | — | Very Long-chain acylCoA Dehydrogenase (VLCAD) Deficiency | No_Longer_Available | — | — | ClinicalTrials.gov |
| NCT07511556 | First-in-human Study of UX016 in GNEM | Phase1 | GNE Myopathy | Not_Yet_Recruiting | 2026-10-01 | 2028-12-01 | ClinicalTrials.gov |
| NCT06067802 | Study of Triheptanoin for the Prevention of Hypoglycemia in Patients With Mediu… | Phase2 | Medium-chain Acyl-CoA Dehydrogenase Deficiency | Withdrawn | 2026-08-01 | 2029-03-01 | ClinicalTrials.gov |
| NCT07097311 | Study to Evaluate the Use of Triheptanoin in Patients With Medium Chain Acyl-Co… | Phase2 | Medium-chain Acyl-CoA Dehydrogenase Deficiency | Withdrawn | 2026-07-01 | 2028-04-01 | ClinicalTrials.gov |
| NCT07447648 | Assessing the Impact of Intensification of Lipid Lowering Therapy With Guidelin… | — | Homozygous Familial Hypercholesterolemia (HoFH) | Recruiting | 2026-04-30 | 2029-02-01 | ClinicalTrials.gov |
| NCT07157254 | A Safety and Efficacy Study of GTX-102 in Subjects With Deletion- or Nondeletio… | Phase2 | Angelman Syndrome | Recruiting | 2025-10-13 | 2030-01-01 | ClinicalTrials.gov |
| NCT07159581 | Gene Therapy for Wilson Disease Evaluated by 64Cu PET/CT | — | Wilson Disease | Enrolling_By_Invitation | 2025-07-08 | 2026-01-01 | ClinicalTrials.gov |
| NCT06617429 | Phase 3 Efficacy and Safety Study of GTX-102 in Pediatric Subjects With Angelma… | Phase3 | Angelman Syndrome | Active_Not_Recruiting | 2024-12-03 | 2027-11-01 | ClinicalTrials.gov |
| NCT06636383 | Glycogen Storage Disease Type Ia (GSDIa) Disease Monitoring Program | — | Glycogen Storage Disease Type Ia | Recruiting | 2024-11-04 | 2036-12-01 | ClinicalTrials.gov |
| NCT06636071 | Setrusumab in Pediatric Japanese Subjects With Osteogenesis Imperfecta | Phase3 | Osteogenesis Imperfecta | Active_Not_Recruiting | 2024-10-25 | 2028-01-01 | ClinicalTrials.gov |
| NCT06415344 | Long-term Extension of GTX-102 in Angelman Syndrome | Phase3 | Angelman Syndrome | Enrolling_By_Invitation | 2024-07-31 | 2029-02-01 | ClinicalTrials.gov |
| NCT06340685 | Triheptanoin for Children With Primary-Specific Pyruvate Dehydrogenase Complex … | Phase1 | Pyruvate Dehydrogenase Complex Deficiency | Recruiting | 2024-07-11 | 2029-06-30 | ClinicalTrials.gov |
| NCT05768854 | Setrusumab vs Bisphosphonates in Pediatric Subjects With Osteogenesis Imperfecta | Phase3 | Osteogenesis Imperfecta | Active_Not_Recruiting | 2023-06-14 | 2027-04-01 | ClinicalTrials.gov |
| NCT05783791 | Development of a Newborn Screening Assay for Angelman Syndrome and Prader-Willi… | — | Angelman Syndrome | Completed | 2023-04-20 | 2023-07-21 | ClinicalTrials.gov |
| NCT05933200 | A Study to Determine the Effect of Triheptanoin Compared With Even-Chain MCT on… | Phase3 | Long-chain Fatty Acid Oxidation Disorders (LC-FAOD) | Active_Not_Recruiting | 2023-02-28 | 2027-08-01 | ClinicalTrials.gov |
| NCT05611528 | Safety and Effectiveness of Evinacumab for the Treatment of Homozygous Familial… | Phase3 | Homozygous Familial Hypercholesterolemia | Completed | 2023-02-21 | 2025-03-18 | ClinicalTrials.gov |
| NCT05345171 | Clinical Study of DTX301 AAV-Mediated Gene Transfer for Ornithine Transcarbamyl… | Phase3 | OTC Deficiency | Active_Not_Recruiting | 2022-10-18 | 2031-03-01 | ClinicalTrials.gov |
| NCT04812106 | Long-Chain Fatty Acid Oxidation Disorders Online Disease Monitoring Program | — | Long-chain Fatty Acid Oxidation Disorders (LC-FAOD) | Terminated | 2022-07-25 | 2022-10-27 | ClinicalTrials.gov |
| NCT05196165 | Clinical Survey Study to Assess Physical Function and the Incidence of Hypoglyc… | — | Glycogen Storage Disease Type III | Terminated | 2022-05-20 | 2023-03-02 | ClinicalTrials.gov |
| NCT05312697 | Long-term Extension Study of Setrusumab in Adults With Type I, III, or IV Osteo… | Phase2 | Osteogenesis Imperfecta | Terminated | 2022-04-28 | 2022-07-07 | ClinicalTrials.gov |
| NCT05125809 | Setrusumab vs Placebo for Osteogenesis Imperfecta | Phase2 | Osteogenesis Imperfecta | Active_Not_Recruiting | 2022-02-21 | 2027-04-01 | ClinicalTrials.gov |
| NCT04783428 | Tumor-induced Osteomalacia Disease Monitoring Program | — | Tumor-induced Osteomalacia (TIO) | Active_Not_Recruiting | 2022-01-31 | 2032-02-28 | ClinicalTrials.gov |
| NCT04632953 | Long-Chain Fatty Acid Oxidation Disorders In-Clinic Disease Monitoring Program | — | Long-chain Fatty Acid Oxidation Disorders (LC-FAOD) | Active_Not_Recruiting | 2021-11-30 | 2035-12-01 | ClinicalTrials.gov |
| NCT05139316 | A Study of Adeno-Associated Virus Serotype 8-Mediated Gene Transfer of Glucose-… | Phase3 | Glycogen Storage Disease Type IA | Completed | 2021-11-08 | 2026-02-20 | ClinicalTrials.gov |
| NCT04990388 | Safety, Tolerability, and Pharmacokinetics of UX053 in Patients With Glycogen S… | Phase1 | Glycogen Storage Disease Type III | Terminated | 2021-10-18 | 2023-03-20 | ClinicalTrials.gov |
| NCT05063435 | Cardiovascular Structure and Function in the Mucopolysaccharidoses | — | Mucopolysaccharidoses | Active_Not_Recruiting | 2021-10-13 | 2026-06-30 | ClinicalTrials.gov |
| NCT04884815 | A Phase 1/2/3 Study of UX701 Gene Therapy in Adults With Wilson Disease | Phase1 | Wilson Disease | Active_Not_Recruiting | 2021-09-27 | 2034-03-01 | ClinicalTrials.gov |
| NCT04909346 | Adeno-Associated Virus (AAV) Antibody Study in Subjects OTC Deficiency, GSDIa, … | — | Ornithine Transcarbamylase Deficiency | Terminated | 2021-06-23 | 2022-11-17 | ClinicalTrials.gov |
| NCT04311307 | Endogenous Glucose Production in Patients With Glycogen Storage Disease Type Ia | Phase1 | Glycogen Storage Disease Type IA | Completed | 2021-01-19 | 2022-02-07 | ClinicalTrials.gov |
| NCT04531189 | Clinical Evaluation and Assessment of Instruments and Biomarkers in Subjects Wi… | — | Wilson Disease | Completed | 2020-12-11 | 2022-03-25 | ClinicalTrials.gov |
| NCT04574830 | Study to Evaluate Biomarkers and Clinical Manifestations in Individuals With Gl… | — | Glycogen Storage Disease Type III | Completed | 2020-11-23 | 2022-06-30 | ClinicalTrials.gov |
| NCT04708015 | Retrospective Study of Glucose Monitoring for Glycemic Control in Patients With… | — | Glycogen Storage Disease Type IA | Completed | 2020-11-05 | 2022-03-04 | ClinicalTrials.gov |
| NCT03665636 | Anaplerotic Therapy Using Triheptanoin for Patients With Glycogen Storage Disea… | Early_Phase1 | Glycogen Storage Disease Type I | Completed | 2020-10-16 | 2021-10-21 | ClinicalTrials.gov |
| NCT04717453 | Study to Characterize Rate of Ureagenesis in Patients With Ornithine Transcarba… | — | Ornithine Transcarbamylase Deficiency | Terminated | 2020-10-06 | 2021-12-15 | ClinicalTrials.gov |
| NCT04360265 | Follow-up Study of AAV-Mediated Gene Transfer (UX111; Previously Known as ABO-1… | Phase3 | Mucopolysaccharidosis IIIA | Enrolling_By_Invitation | 2020-09-28 | 2027-08-01 | ClinicalTrials.gov |
| NCT04320316 | A Trial to Assess the Safety and Efficacy of KRN23 in Epidermal Nevus Syndrome … | Phase4 | Epidermal Nevus Syndrome | Completed | 2020-07-31 | 2021-08-31 | ClinicalTrials.gov |
| NCT04259281 | A Study of the Safety and Tolerability of GTX-102 in Children With Angelman Syn… | Phase1 | Angelman Syndrome | Completed | 2020-02-24 | 2025-01-08 | ClinicalTrials.gov |
| NCT04088734 | Gene Transfer Study of ABO-102 in Patients With Middle and Advanced Phases of M… | Phase1 | MPS IIIA | Terminated | 2019-09-18 | 2022-03-10 | ClinicalTrials.gov |
| NCT04269122 | A Study to Assess Plasma Ammonia Time-Normalized Area Under the Curve and Rate … | — | Ornithine Transcarbamylase Deficiency | Completed | 2019-08-02 | 2020-02-20 | ClinicalTrials.gov |
| NCT03970278 | Study of Long-Term Safety and Efficacy on Gene Therapy in Glycogen Storage Dise… | — | Glycogen Storage Disease Type IA | Completed | 2019-07-15 | 2025-02-25 | ClinicalTrials.gov |
| NCT03993821 | Burosumab for CSHS | Early_Phase1 | Cutaneous Skeletal Hypophosphatemia Syndrome (CSHS) | Unknown | 2019-07-01 | 2023-03-01 | ClinicalTrials.gov |
| NCT03768817 | Clinical Outcome of Triheptanoin Treatment in Patients With Long-chain Fatty Ac… | — | Long-chain Fatty Acid Oxidation Disorders (LC-FAOD) | Completed | 2019-01-30 | 2020-06-08 | ClinicalTrials.gov |
| NCT03636438 | Long Term Follow Up to Evaluate DTX301 in Adults With Late-Onset OTC Deficiency | — | Ornithine Transcarbamylase (OTC) Deficiency | Active_Not_Recruiting | 2018-08-30 | 2029-12-01 | ClinicalTrials.gov |
| NCT03642860 | The Effect of Triheptanoin on Fatty Acid Oxidation and Exercise Tolerance in Pa… | Phase2 | Tarui Disease | Completed | 2018-08-15 | 2019-08-28 | ClinicalTrials.gov |
| NCT03651505 | X-linked Hypophosphatemia Disease Monitoring Program | — | X-linked Hypophosphatemia | Active_Not_Recruiting | 2018-07-16 | 2032-12-01 | ClinicalTrials.gov |
| NCT03506425 | A Pilot Trial of Triheptanoin for People With Amyotrophic Lateral Sclerosis (PA… | Phase1 | ALS | Completed | 2018-06-21 | 2019-03-28 | ClinicalTrials.gov |
| NCT03517085 | Safety and Dose-Finding Study of DTX401 (AAV8G6PC) in Adults With Glycogen Stor… | Phase1 | GSD1 | Completed | 2018-05-18 | 2021-11-02 | ClinicalTrials.gov |
| NCT03581591 | Open Label Trial Assessing Safety and Efficacy of Burosumab (KRN23), in a Patie… | Phase3 | Hypophosphatemia | Completed | 2018-01-31 | 2019-12-06 | ClinicalTrials.gov |
| NCT03604835 | Mucopolysaccharidosis VII Disease Monitoring Program | — | Mucopolysaccharidosis VII | Recruiting | 2018-01-29 | 2032-04-01 | ClinicalTrials.gov |
| NCT03216486 | An Exploratory Study of BPS804 Treatment in Adult Patients With Type I, III or … | Phase2 | Osteogenesis Imperfecta | Withdrawn | 2017-10-31 | 2018-11-01 | ClinicalTrials.gov |
| NCT03118570 | A Study in Adult Patients With Type I, III or IV Osteogenesis Imperfecta Treate… | Phase2 | Osteogenesis Imperfecta, Type I | Completed | 2017-09-11 | 2020-11-12 | ClinicalTrials.gov |
| NCT02991144 | Safety and Dose-Finding Study of DTX301 (scAAV8OTC) in Adults With Late-Onset O… | Phase1 | Ornithine Transcarbamylase (OTC) Deficiency | Completed | 2017-07-31 | 2021-12-16 | ClinicalTrials.gov |
| NCT02960217 | Crossover Study to Assess the Efficacy and Safety of UX007 in the Treatment of … | Phase3 | Glucose Transporter Type 1 Deficiency Syndrome (Glut1 DS) | Terminated | 2017-04-19 | 2019-10-09 | ClinicalTrials.gov |
| NCT03059160 | Open Label Trial of Triheptanoin (UX007) in Treatment of Rett Syndrome. | Phase2 | Rett Syndrome | Unknown | 2017-04-01 | 2018-08-01 | ClinicalTrials.gov |
| NCT02971969 | Long-Term Safety, Tolerability, and Efficacy of DTX101 (AAVrh10FIX) in Adults W… | — | Hemophilia B | Completed | 2017-01-01 | 2021-11-06 | ClinicalTrials.gov |
| NCT02931682 | Observational Study of Males With Creatine Transporter Deficiency | — | Creatine Deficiency, X-linked | Terminated | 2016-12-01 | 2022-10-24 | ClinicalTrials.gov |
| NCT02696044 | Treatment of Mitochondrial Dysfunction in Rett Syndrome With Triheptanoin | Phase2 | Rett Syndrome | Unknown | 2016-06-01 | 2022-02-01 | ClinicalTrials.gov |
| NCT02736188 | Study to Evaluate the Safety and Efficacy of Aceneuramic Acid Extended-Release … | Phase3 | Hereditary Inclusion Body Myopathy | Terminated | 2016-05-02 | 2018-01-10 | ClinicalTrials.gov |
| NCT02731690 | A Study to Evaluate the Safety of Aceneuramic Acid Extended Release (Ace-ER; UX… | Phase2 | Hereditary Inclusion Body Myopathy | Terminated | 2016-04-29 | 2018-01-10 | ClinicalTrials.gov |
| NCT02716246 | Phase I/II/III Gene Transfer Clinical Trial of scAAV9.U1a.hSGSH | Phase2 | MPS IIIA | Recruiting | 2016-04-25 | 2029-03-01 | ClinicalTrials.gov |
| NCT02679235 | Brain Energy and Aging With Triheptanoin | Phase1 | Frontal Lobe Hypometabolism | Completed | 2016-04-01 | 2018-08-08 | ClinicalTrials.gov |
| NCT02618915 | Safety and Dose Finding Study of DTX101 (AAVrh10FIX) in Adults With Moderate/Se… | Phase1 | Hemophilia B | Terminated | 2015-12-16 | 2017-10-18 | ClinicalTrials.gov |
| NCT02432144 | A Study of UX003 Recombinant Human Beta-Glucuronidase (rhGUS) Enzyme Replacemen… | Phase3 | Sly Syndrome | Completed | 2015-11-10 | 2019-01-14 | ClinicalTrials.gov |
| NCT02599961 | Study to Assess the Long Term Safety and Efficacy of UX007 in Participants With… | Phase2 | Glucose Transporter Type 1 Deficiency Syndrome | Terminated | 2015-09-10 | 2019-10-22 | ClinicalTrials.gov |
| NCT02418455 | Study of UX003 Recombinant Human Beta-Glucuronidase (rhGUS) Enzyme Replacement … | Phase2 | Sly Syndrome | Completed | 2015-07-21 | 2019-03-26 | ClinicalTrials.gov |
| NCT02453061 | A Comparative Phase 2 Study Assessing the Efficacy of Triheptanoin, an Anaplero… | Phase2 | Huntington Disease | Completed | 2015-06-29 | 2019-12-03 | ClinicalTrials.gov |
| NCT02377921 | Phase 3 Randomized, Double-Blind, Placebo-Controlled Study to Evaluate Sialic A… | Phase3 | Hereditary Inclusion Body Myopathy | Completed | 2015-05-20 | 2017-06-09 | ClinicalTrials.gov |
| NCT02432768 | The Effect of Triheptanoin in Adults With McArdle Disease (Glycogen Storage Dis… | Phase2 | Glycogen Storage Disease Type V | Completed | 2015-04-01 | 2019-02-01 | ClinicalTrials.gov |
| NCT02214160 | Long-Chain Fatty Acid Oxidation Disorders (LC-FAOD) Extension Study for Subject… | Phase2 | Carnitine Palmitoyltransferase (CPT I or CPT II) Deficiency | Completed | 2014-12-09 | 2020-12-03 | ClinicalTrials.gov |
| NCT02230566 | A Phase 3 Study of UX003 Recombinant Human Betaglucuronidase (rhGUS) Enzyme Rep… | Phase3 | MPS 7 | Completed | 2014-12-01 | 2016-05-01 | ClinicalTrials.gov |
| NCT02000960 | Pilot Study of Triheptanoin in Patients With Glucose Transporter 1 Deficiency S… | Phase2 | Glucose Transporter Type 1 Deficiency Syndrome | Unknown | 2014-04-01 | 2016-10-01 | ClinicalTrials.gov |
| NCT01993186 | Phase 2 Study of Triheptanoin (UX007) for the Treatment of Glucose Transporter … | Phase2 | Glucose Transporter Type 1 Deficiency Syndrome (Glut1 DS) | Completed | 2014-02-28 | 2017-09-20 | ClinicalTrials.gov |
| NCT02036853 | An Open-Label Trial of Triheptanoin in Patients With Glucose Transporter Type-1… | Phase2 | Glucose Transporter Type-1 Deficiency Syndrome (Glut1 DS) | Completed | 2014-02-20 | 2019-06-30 | ClinicalTrials.gov |
| NCT01886378 | A Study of UX007 (Triheptanoin) in Participants With Long-Chain Fatty Acid Oxid… | Phase2 | Long-chain Fatty Acid Oxidation Disorders (LC-FAOD) | Completed | 2014-02-06 | 2016-08-25 | ClinicalTrials.gov |
| NCT02014883 | Phase II Open Label Study Using Triheptanoin in Patients With Glucose Type 1 Tr… | Phase2 | Glut1 Deficiency Syndrome | Completed | 2013-12-04 | 2019-07-04 | ClinicalTrials.gov |
| NCT01856218 | An Open-Label Phase 1/2 Study to Assess the Safety, Efficacy and Dose of Study … | Phase1 | Mucopolysaccharidosis Type 7 | Completed | 2013-11-01 | 2016-07-01 | ClinicalTrials.gov |
| NCT01806610 | Study of Safety and Tolerability of BPS804 in Patients With Late-stage Chronic … | Phase2 | Chronic-kidney Disease Stage 5D on Stable Hemodialysis | Withdrawn | 2013-08-01 | 2014-04-01 | ClinicalTrials.gov |
| NCT01830972 | An Open Label Phase 2 Extension Study of Higher Dose Sialic Acid-Extended Relea… | Phase2 | GNE Myopathy | Completed | 2013-06-04 | 2017-02-14 | ClinicalTrials.gov |
| NCT01784679 | GNE-Myopathy Disease Monitoring Program (GNEM-DMP): A Registry and Prospective … | — | Hereditary Inclusion Body Myopathy | Completed | 2013-04-05 | 2017-11-30 | ClinicalTrials.gov |
| NCT01517880 | A Phase 2 Study to Evaluate the Dose and Pharmacodynamic Efficacy of Sialic Aci… | Phase2 | GNE Myopathy | Completed | 2012-05-01 | 2013-11-01 | ClinicalTrials.gov |
| NCT01406548 | Safety and Efficacy of Multiple Dosing Regimens of BPS804 in Post Menopausal Wo… | Phase2 | Osteopenia | Completed | 2011-07-01 | 2013-10-01 | ClinicalTrials.gov |
| NCT01406977 | Dose Escalation Study to Evaluate the Safety and Tolerability of Multiple Infus… | Phase2 | Hypophosphatasia | Completed | 2011-07-01 | 2012-09-01 | ClinicalTrials.gov |
| NCT01359319 | Safety and Pharmacokinetics of Sialic Acid Tables in Patients With Hereditary I… | Phase1 | Hereditary Inclusion Body Myopathy (HIBM) | Completed | 2011-07-01 | 2012-05-01 | ClinicalTrials.gov |
| NCT01417091 | Safety, Pharmacokinetics and Pharmacodynamics of BPS804 in Osteogenesis Imperfe… | Phase2 | Osteogenesis Imperfecta | Completed | 2011-06-01 | 2012-12-01 | ClinicalTrials.gov |
| NCT00947960 | Triheptanoin Treatment Trial for Patients With Adult Polyglucosan Body Disease | Phase2 | Adult Polyglucosan Body Disease | Completed | 2009-06-01 | 2015-07-01 | ClinicalTrials.gov |
Total clinical trials: 89
| Product Name | Type | Development Stage | Therapeutic Area | Study Status | Trial ID |
|---|---|---|---|---|---|
| No Intervention | Other | Preclinical | Glycogen Storage Disease Type Ia | RECRUITING | NCT06636383 |
| Triheptanoin | Other | Phase PHASE2 | Medium-chain Acyl-CoA Dehydrogenase Deficiency | WITHDRAWN | NCT06067802 |
| Triheptanoin | Other | Phase PHASE2 | Medium-chain Acyl-CoA Dehydrogenase Deficiency | WITHDRAWN | NCT07097311 |
| GTX-102 | Other | Phase PHASE2 | Angelman Syndrome | RECRUITING | NCT07157254 |
| No intervention | Other | Phase PHASE2 | Angelman Syndrome | RECRUITING | NCT07157254 |
| setrusumab | Other | Phase PHASE3 | Osteogenesis Imperfecta | ACTIVE_NOT_RECRUITING | NCT06636071 |
| Standard of Care (SOC) | Other | Phase PHASE1 | Wilson Disease | ACTIVE_NOT_RECRUITING | NCT04884815 |
| UX701 | Other | Phase PHASE1 | Wilson Disease | ACTIVE_NOT_RECRUITING | NCT04884815 |
| Adjuvant IM Therapy | Drug | Phase PHASE2 | MPS IIIA | RECRUITING | NCT02716246 |
| Optimized Prophylactic IM Therapy | Drug | Phase PHASE2 | MPS IIIA | RECRUITING | NCT02716246 |
| Prophylactic Immunomodulatory (IM) Therapy | Drug | Phase PHASE2 | MPS IIIA | RECRUITING | NCT02716246 |
| UX111 | Other | Phase PHASE2 | MPS IIIA | RECRUITING | NCT02716246 |
| No Intervention | Other | Preclinical | Mucopolysaccharidosis VII | RECRUITING | NCT03604835 |
| GTX-102 | Other | Phase PHASE2 | Angelman Syndrome | RECRUITING | NCT07157254 |
| No intervention | Other | Phase PHASE2 | Angelman Syndrome | RECRUITING | NCT07157254 |
| GTX-102 | Other | Phase PHASE2 | Angelman Syndrome | RECRUITING | NCT07157254 |
| No intervention | Other | Phase PHASE2 | Angelman Syndrome | RECRUITING | NCT07157254 |
| Placebo | Other | Phase PHASE2 | Huntington Disease | COMPLETED | NCT02453061 |
| Triheptanoin oil | Other | Phase PHASE2 | Huntington Disease | COMPLETED | NCT02453061 |
| No intervention | Other | Preclinical | Tumor-induced Osteomalacia (TIO) | ACTIVE_NOT_RECRUITING | NCT04783428 |
| No Intervention | Other | Preclinical | Long-chain Fatty Acid Oxidation Disorders (LC-FAOD) | ACTIVE_NOT_RECRUITING | NCT04632953 |
| GTX-102 | Other | Phase PHASE2 | Angelman Syndrome | RECRUITING | NCT07157254 |
| No intervention | Other | Phase PHASE2 | Angelman Syndrome | RECRUITING | NCT07157254 |
| Sodium Acetate | Other | Phase PHASE3 | OTC Deficiency | ACTIVE_NOT_RECRUITING | NCT05345171 |
| Placebo for oral corticosteroids | Other | Phase PHASE3 | OTC Deficiency | ACTIVE_NOT_RECRUITING | NCT05345171 |
| Oral Corticosteroids | Other | Phase PHASE3 | OTC Deficiency | ACTIVE_NOT_RECRUITING | NCT05345171 |
| Placebo | Other | Phase PHASE3 | OTC Deficiency | ACTIVE_NOT_RECRUITING | NCT05345171 |
| DTX301 | Other | Phase PHASE3 | OTC Deficiency | ACTIVE_NOT_RECRUITING | NCT05345171 |
| No Intervention | Other | Preclinical | Ornithine Transcarbamylase (OTC) Deficiency | ACTIVE_NOT_RECRUITING | NCT03636438 |
| No Intervention | Other | Preclinical | Glycogen Storage Disease Type Ia | RECRUITING | NCT06636383 |
| No intervention | Other | Preclinical | X-linked Hypophosphatemia | ACTIVE_NOT_RECRUITING | NCT03651505 |
| No intervention | Other | Preclinical | X-linked Hypophosphatemia | ACTIVE_NOT_RECRUITING | NCT03651505 |
| No intervention | Other | Preclinical | X-linked Hypophosphatemia | ACTIVE_NOT_RECRUITING | NCT03651505 |
| No intervention | Other | Preclinical | X-linked Hypophosphatemia | ACTIVE_NOT_RECRUITING | NCT03651505 |
| No intervention | Other | Preclinical | X-linked Hypophosphatemia | ACTIVE_NOT_RECRUITING | NCT03651505 |
| Sham-LP | Other | Phase PHASE3 | Angelman Syndrome | ACTIVE_NOT_RECRUITING | NCT06617429 |
| GTX-102 | Other | Phase PHASE3 | Angelman Syndrome | ACTIVE_NOT_RECRUITING | NCT06617429 |
| GTX-102 | Other | Phase PHASE3 | Angelman Syndrome | ENROLLING_BY_INVITATION | NCT06415344 |
| Sham-LP | Other | Phase PHASE3 | Angelman Syndrome | ACTIVE_NOT_RECRUITING | NCT06617429 |
| GTX-102 | Other | Phase PHASE3 | Angelman Syndrome | ACTIVE_NOT_RECRUITING | NCT06617429 |
| GTX-102 | Other | Phase PHASE3 | Angelman Syndrome | ENROLLING_BY_INVITATION | NCT06415344 |
| Sham-LP | Other | Phase PHASE3 | Angelman Syndrome | ACTIVE_NOT_RECRUITING | NCT06617429 |
| GTX-102 | Other | Phase PHASE3 | Angelman Syndrome | ACTIVE_NOT_RECRUITING | NCT06617429 |
| GTX-102 | Other | Phase PHASE3 | Angelman Syndrome | ENROLLING_BY_INVITATION | NCT06415344 |
| Sham-LP | Other | Phase PHASE3 | Angelman Syndrome | ACTIVE_NOT_RECRUITING | NCT06617429 |
| GTX-102 | Other | Phase PHASE3 | Angelman Syndrome | ACTIVE_NOT_RECRUITING | NCT06617429 |
| GTX-102 | Other | Phase PHASE3 | Angelman Syndrome | ENROLLING_BY_INVITATION | NCT06415344 |
| Sham-LP | Other | Phase PHASE3 | Angelman Syndrome | ACTIVE_NOT_RECRUITING | NCT06617429 |
| GTX-102 | Other | Phase PHASE3 | Angelman Syndrome | ACTIVE_NOT_RECRUITING | NCT06617429 |
| GTX-102 | Other | Phase PHASE3 | Angelman Syndrome | ENROLLING_BY_INVITATION | NCT06415344 |
| Sham-LP | Other | Phase PHASE3 | Angelman Syndrome | ACTIVE_NOT_RECRUITING | NCT06617429 |
| GTX-102 | Other | Phase PHASE3 | Angelman Syndrome | ACTIVE_NOT_RECRUITING | NCT06617429 |
| GTX-102 | Other | Phase PHASE3 | Angelman Syndrome | ENROLLING_BY_INVITATION | NCT06415344 |
| Sham-LP | Other | Phase PHASE3 | Angelman Syndrome | ACTIVE_NOT_RECRUITING | NCT06617429 |
| GTX-102 | Other | Phase PHASE3 | Angelman Syndrome | ACTIVE_NOT_RECRUITING | NCT06617429 |
| GTX-102 | Other | Phase PHASE3 | Angelman Syndrome | ENROLLING_BY_INVITATION | NCT06415344 |
| Sham-LP | Other | Phase PHASE3 | Angelman Syndrome | ACTIVE_NOT_RECRUITING | NCT06617429 |
| GTX-102 | Other | Phase PHASE3 | Angelman Syndrome | ACTIVE_NOT_RECRUITING | NCT06617429 |
| GTX-102 | Other | Phase PHASE3 | Angelman Syndrome | ENROLLING_BY_INVITATION | NCT06415344 |
| Sham-LP | Other | Phase PHASE3 | Angelman Syndrome | ACTIVE_NOT_RECRUITING | NCT06617429 |
| GTX-102 | Other | Phase PHASE3 | Angelman Syndrome | ACTIVE_NOT_RECRUITING | NCT06617429 |
| GTX-102 | Other | Phase PHASE3 | Angelman Syndrome | ENROLLING_BY_INVITATION | NCT06415344 |
| SA-IR 500 mg | Other | Phase PHASE2 | GNE Myopathy | COMPLETED | NCT01830972 |
| SA-ER 500 mg | Other | Phase PHASE2 | GNE Myopathy | COMPLETED | NCT01830972 |
| Triheptanoin | Other | Phase PHASE2 | Glucose Transporter Type-1 Deficiency Syndrome (Glut1 DS) | COMPLETED | NCT02036853 |
| Setrusumab | Other | Phase PHASE2 | Osteogenesis Imperfecta | TERMINATED | NCT05312697 |
| Sialic Acid Extended Release (SA-ER) Tables | Other | Phase PHASE1 | Hereditary Inclusion Body Myopathy (HIBM) | COMPLETED | NCT01359319 |
| Sialic Acid Extended Release (SA-ER) Tablets | Other | Phase PHASE1 | Hereditary Inclusion Body Myopathy (HIBM) | COMPLETED | NCT01359319 |
| Triheptanoin | Other | Phase PHASE2 | Glucose Transporter Type 1 Deficiency Syndrome | UNKNOWN | NCT02000960 |
| Placebo | Other | Phase PHASE2 | GNE Myopathy | COMPLETED | NCT01517880 |
| Sialic Acid Extended Release (SA-ER) | Other | Phase PHASE2 | GNE Myopathy | COMPLETED | NCT01517880 |
| UX003 | Other | Preclinical | Mucopolysaccharidosis Type 7 | NO_LONGER_AVAILABLE | NCT02097251 |
| Tridecanoic Acid | Other | Phase PHASE2 | Rett Syndrome | UNKNOWN | NCT03059160 |
| DTX101 | Other | Phase PHASE1 | Hemophilia B | TERMINATED | NCT02618915 |
| UX003 | Other | Phase PHASE1 | Mucopolysaccharidosis Type 7 | COMPLETED | NCT01856218 |
| Triheptanoin | Other | Preclinical | Glucose Transporter 1 Deficiency Syndrome | NO_LONGER_AVAILABLE | NCT02968953 |
| Aceneuramic Acid Extended-Release | Other | Phase PHASE2 | Hereditary Inclusion Body Myopathy | TERMINATED | NCT02731690 |
| Placebo | Other | Phase PHASE3 | Hereditary Inclusion Body Myopathy | COMPLETED | NCT02377921 |
| aceneuramic acid extended-release (Ace-ER) | Other | Phase PHASE3 | Hereditary Inclusion Body Myopathy | COMPLETED | NCT02377921 |
| Placebo oil | Other | Phase PHASE2 | Glycogen Storage Disease Type V | COMPLETED | NCT02432768 |
| Triheptanoin | Other | Phase PHASE2 | Glycogen Storage Disease Type V | COMPLETED | NCT02432768 |
| UX003 | Other | Phase PHASE2 | Sly Syndrome | COMPLETED | NCT02418455 |
| Triheptanoin | Other | Phase PHASE1 | ALS | COMPLETED | NCT03506425 |
| Burosumab | Other | Phase PHASE3 | Hypophosphatemia | COMPLETED | NCT03581591 |
| No Intervention | Other | Preclinical | Ornithine Transcarbamylase Deficiency | COMPLETED | NCT04269122 |
| UX007 | Other | Phase PHASE2 | Glucose Transporter Type 1 Deficiency Syndrome | TERMINATED | NCT02599961 |
| Placebo | Other | Phase PHASE3 | Glucose Transporter Type 1 Deficiency Syndrome (Glut1 DS) | TERMINATED | NCT02960217 |
| UX007 | Other | Phase PHASE3 | Glucose Transporter Type 1 Deficiency Syndrome (Glut1 DS) | TERMINATED | NCT02960217 |
| Placebo | Other | Phase PHASE2 | Glucose Transporter Type 1 Deficiency Syndrome (Glut1 DS) | COMPLETED | NCT01993186 |
| UX007 | Other | Phase PHASE2 | Glucose Transporter Type 1 Deficiency Syndrome (Glut1 DS) | COMPLETED | NCT01993186 |
| triheptanoin | Other | Phase PHASE2 | Rett Syndrome | UNKNOWN | NCT02696044 |
| POST Triheptanoin | Other | Phase PHASE1 | Frontal Lobe Hypometabolism | COMPLETED | NCT02679235 |
| UX003 | Other | Phase PHASE3 | Sly Syndrome | COMPLETED | NCT02432144 |
| Placebo | Other | Phase PHASE3 | MPS 7 | COMPLETED | NCT02230566 |
| UX003 | Other | Phase PHASE3 | MPS 7 | COMPLETED | NCT02230566 |
| No Intervention | Other | Preclinical | Long-chain Fatty Acid Oxidation Disorders (LC-FAOD) | COMPLETED | NCT03768817 |
| UX007 | Other | Phase PHASE2 | Long-chain Fatty Acid Oxidation Disorders (LC-FAOD) | COMPLETED | NCT01886378 |
| BPS804 | Other | Phase PHASE2 | Osteogenesis Imperfecta | COMPLETED | NCT01417091 |
| triheptanoin | Other | Preclinical | Very Long-chain acylCoA Dehydrogenase (VLCAD) Deficiency | NO_LONGER_AVAILABLE | NCT01461304 |
| Triheptanoin | Other | Phase EARLY_PHASE1 | Glycogen Storage Disease Type I | COMPLETED | NCT03665636 |
| [6,6-2H2]glucose | Other | Phase PHASE1 | Glycogen Storage Disease Type IA | COMPLETED | NCT04311307 |
| No Intervention | Other | Preclinical | Ornithine Transcarbamylase Deficiency | TERMINATED | NCT04717453 |
| Burosumab | Other | Phase EARLY_PHASE1 | Cutaneous Skeletal Hypophosphatemia Syndrome (CSHS) | UNKNOWN | NCT03993821 |
| No Intervention | Other | Preclinical | Glycogen Storage Disease Type III | COMPLETED | NCT04574830 |
| Placebo | Other | Phase PHASE2 | Chronic-kidney Disease Stage 5D on Stable Hemodialysis | WITHDRAWN | NCT01806610 |
| BPS804 | Other | Phase PHASE2 | Chronic-kidney Disease Stage 5D on Stable Hemodialysis | WITHDRAWN | NCT01806610 |
| Placebo to 20mg/Kg BPS804 | Other | Phase PHASE2 | Osteopenia | COMPLETED | NCT01406548 |
| BPS804 20mg/Kg | Other | Phase PHASE2 | Osteopenia | COMPLETED | NCT01406548 |
| BPS804 | Other | Phase PHASE2 | Hypophosphatasia | COMPLETED | NCT01406977 |
| Crysvita (burosumab-twza) Treatment | Drug | Phase PHASE4 | Epidermal Nevus Syndrome | COMPLETED | NCT04320316 |
| No Intervention | Other | Preclinical | Long-chain Fatty Acid Oxidation Disorders (LC-FAOD) | TERMINATED | NCT04812106 |
| steroid regimen | Other | Phase PHASE1 | GSD1 | COMPLETED | NCT03517085 |
| DTX401 | Other | Phase PHASE1 | GSD1 | COMPLETED | NCT03517085 |
| Prophylactic Corticosteroid Taper Regimen | Other | Phase PHASE1 | Ornithine Transcarbamylase (OTC) Deficiency | COMPLETED | NCT02991144 |
| Reactive Corticosteroid Taper Regimen | Other | Phase PHASE1 | Ornithine Transcarbamylase (OTC) Deficiency | COMPLETED | NCT02991144 |
| scAAV8OTC | Other | Phase PHASE1 | Ornithine Transcarbamylase (OTC) Deficiency | COMPLETED | NCT02991144 |
| Aceneuramic Acid Extended-Release Tablets | Other | Phase PHASE3 | Hereditary Inclusion Body Myopathy | TERMINATED | NCT02736188 |
| BPS804 | Other | Phase PHASE2 | Osteogenesis Imperfecta | WITHDRAWN | NCT03216486 |
| zoledronic acid (optional) | Other | Phase PHASE2 | Osteogenesis Imperfecta, Type I | COMPLETED | NCT03118570 |
| Vitamin D | Other | Phase PHASE2 | Osteogenesis Imperfecta, Type I | COMPLETED | NCT03118570 |
| Calcium | Other | Phase PHASE2 | Osteogenesis Imperfecta, Type I | COMPLETED | NCT03118570 |
| setrusumab | Other | Phase PHASE2 | Osteogenesis Imperfecta, Type I | COMPLETED | NCT03118570 |
| ABO-102 | Other | Phase PHASE1 | MPS IIIA | TERMINATED | NCT04088734 |
| UX007 | Other | Phase PHASE2 | Carnitine Palmitoyltransferase (CPT I or CPT II) Deficiency | COMPLETED | NCT02214160 |
| Newborn Screening Assay | Other | Preclinical | Angelman Syndrome | COMPLETED | NCT05783791 |
| No Intervention | Other | Preclinical | Glycogen Storage Disease Type III | TERMINATED | NCT05196165 |
| Placebo Oil | Other | Phase PHASE2 | Tarui Disease | COMPLETED | NCT03642860 |
| Triheptanoin | Other | Phase PHASE2 | Tarui Disease | COMPLETED | NCT03642860 |
| H1 Blocker | Other | Phase PHASE1 | Glycogen Storage Disease Type III | TERMINATED | NCT04990388 |
| H2 Blocker | Other | Phase PHASE1 | Glycogen Storage Disease Type III | TERMINATED | NCT04990388 |
| Antipyretic | Other | Phase PHASE1 | Glycogen Storage Disease Type III | TERMINATED | NCT04990388 |
| Placebo | Other | Phase PHASE1 | Glycogen Storage Disease Type III | TERMINATED | NCT04990388 |
| UX053 | Other | Phase PHASE1 | Glycogen Storage Disease Type III | TERMINATED | NCT04990388 |
| No Intervention | Other | Preclinical | Glycogen Storage Disease Type IA | COMPLETED | NCT04708015 |
| Placebo | Other | Phase PHASE2 | Huntington Disease | COMPLETED | NCT02453061 |
| Triheptanoin oil | Other | Phase PHASE2 | Huntington Disease | COMPLETED | NCT02453061 |
| Evinacumab | Other | Phase PHASE3 | Homozygous Familial Hypercholesterolemia | COMPLETED | NCT05611528 |
| Venipuncture | Other | Preclinical | Mucopolysaccharidoses | ACTIVE_NOT_RECRUITING | NCT05063435 |
| Echocardiography, transthoracic | Other | Preclinical | Mucopolysaccharidoses | ACTIVE_NOT_RECRUITING | NCT05063435 |
| Carotid ultrasonography | Other | Preclinical | Mucopolysaccharidoses | ACTIVE_NOT_RECRUITING | NCT05063435 |
| Triheptanoin | Other | Preclinical | Long Chain Fatty Acid Oxidation Disorders | AVAILABLE | NCT03773770 |
| No Intervention | Other | Preclinical | Glycogen Storage Disease Type Ia | RECRUITING | NCT06636383 |
| GLUT1 DS | Other | Phase PHASE2 | Glut1 Deficiency Syndrome | COMPLETED | NCT02014883 |
| GTX-102 | Other | Phase PHASE3 | Angelman Syndrome | ENROLLING_BY_INVITATION | NCT06415344 |
| GTX-102 | Other | Phase PHASE1 | Angelman Syndrome | COMPLETED | NCT04259281 |
| No intervention | Other | Preclinical | Tumor-induced Osteomalacia (TIO) | ACTIVE_NOT_RECRUITING | NCT04783428 |
| Sham-LP | Other | Phase PHASE3 | Angelman Syndrome | ACTIVE_NOT_RECRUITING | NCT06617429 |
| GTX-102 | Other | Phase PHASE3 | Angelman Syndrome | ACTIVE_NOT_RECRUITING | NCT06617429 |
| Triheptanoin | Other | Phase PHASE1 | Pyruvate Dehydrogenase Complex Deficiency | RECRUITING | NCT06340685 |
| Sodium Acetate | Other | Phase PHASE3 | OTC Deficiency | ACTIVE_NOT_RECRUITING | NCT05345171 |
| Placebo for oral corticosteroids | Other | Phase PHASE3 | OTC Deficiency | ACTIVE_NOT_RECRUITING | NCT05345171 |
| Oral Corticosteroids | Other | Phase PHASE3 | OTC Deficiency | ACTIVE_NOT_RECRUITING | NCT05345171 |
| Placebo | Other | Phase PHASE3 | OTC Deficiency | ACTIVE_NOT_RECRUITING | NCT05345171 |
| DTX301 | Other | Phase PHASE3 | OTC Deficiency | ACTIVE_NOT_RECRUITING | NCT05345171 |
| No intervention | Other | Preclinical | X-linked Hypophosphatemia | ACTIVE_NOT_RECRUITING | NCT03651505 |
| No Intervention | Other | Preclinical | Ornithine Transcarbamylase (OTC) Deficiency | ACTIVE_NOT_RECRUITING | NCT03636438 |
| Setrusumab | Other | Phase PHASE3 | Osteogenesis Imperfecta | ACTIVE_NOT_RECRUITING | NCT05768854 |
| Bisphosphonate | Other | Phase PHASE3 | Osteogenesis Imperfecta | ACTIVE_NOT_RECRUITING | NCT05768854 |
| Placebo | Other | Phase PHASE2 | Osteogenesis Imperfecta | ACTIVE_NOT_RECRUITING | NCT05125809 |
| Setrusumab | Other | Phase PHASE2 | Osteogenesis Imperfecta | ACTIVE_NOT_RECRUITING | NCT05125809 |
| No Intervention | Other | Preclinical | Long-chain Fatty Acid Oxidation Disorders (LC-FAOD) | ACTIVE_NOT_RECRUITING | NCT04632953 |
| setrusumab | Other | Phase PHASE3 | Osteogenesis Imperfecta | ACTIVE_NOT_RECRUITING | NCT06636071 |
| Standard of Care (SOC) | Other | Phase PHASE1 | Wilson Disease | ACTIVE_NOT_RECRUITING | NCT04884815 |
| UX701 | Other | Phase PHASE1 | Wilson Disease | ACTIVE_NOT_RECRUITING | NCT04884815 |
| No intervention | Other | Preclinical | Glycogen Storage Disease Type IA | COMPLETED | NCT03970278 |
| Placebo for oral prednisolone | Other | Phase PHASE3 | Glycogen Storage Disease Type IA | COMPLETED | NCT05139316 |
| Oral prednisolone | Other | Phase PHASE3 | Glycogen Storage Disease Type IA | COMPLETED | NCT05139316 |
| Placebo | Other | Phase PHASE3 | Glycogen Storage Disease Type IA | COMPLETED | NCT05139316 |
| DTX401 | Other | Phase PHASE3 | Glycogen Storage Disease Type IA | COMPLETED | NCT05139316 |
| MCT Oil | Other | Phase PHASE3 | Long-chain Fatty Acid Oxidation Disorders (LC-FAOD) | ACTIVE_NOT_RECRUITING | NCT05933200 |
| Triheptanoin | Other | Phase PHASE3 | Long-chain Fatty Acid Oxidation Disorders (LC-FAOD) | ACTIVE_NOT_RECRUITING | NCT05933200 |
| Mepsevii | Other | Preclinical | MPS VII | AVAILABLE | NCT03775174 |
| No Intervention | Other | Preclinical | Mucopolysaccharidosis VII | RECRUITING | NCT03604835 |
| GTX-102 | Other | Phase PHASE2 | Angelman Syndrome | RECRUITING | NCT07157254 |
| No intervention | Other | Phase PHASE2 | Angelman Syndrome | RECRUITING | NCT07157254 |
| Adjuvant Immunomodulatory (IM) Therapy | Drug | Phase PHASE3 | Mucopolysaccharidosis IIIA | ENROLLING_BY_INVITATION | NCT04360265 |
| No Investigational Product | Other | Phase PHASE3 | Mucopolysaccharidosis IIIA | ENROLLING_BY_INVITATION | NCT04360265 |
| Adjuvant IM Therapy | Drug | Phase PHASE2 | MPS IIIA | RECRUITING | NCT02716246 |
| Optimized Prophylactic IM Therapy | Drug | Phase PHASE2 | MPS IIIA | RECRUITING | NCT02716246 |
| Prophylactic Immunomodulatory (IM) Therapy | Drug | Phase PHASE2 | MPS IIIA | RECRUITING | NCT02716246 |
| UX111 | Other | Phase PHASE2 | MPS IIIA | RECRUITING | NCT02716246 |
| GTX-102 | Other | Phase PHASE2 | Angelman Syndrome | RECRUITING | NCT07157254 |
| No intervention | Other | Phase PHASE2 | Angelman Syndrome | RECRUITING | NCT07157254 |
| Adjuvant IM Therapy | Drug | Phase PHASE2 | MPS IIIA | RECRUITING | NCT02716246 |
| Optimized Prophylactic IM Therapy | Drug | Phase PHASE2 | MPS IIIA | RECRUITING | NCT02716246 |
| Prophylactic Immunomodulatory (IM) Therapy | Drug | Phase PHASE2 | MPS IIIA | RECRUITING | NCT02716246 |
| UX111 | Other | Phase PHASE2 | MPS IIIA | RECRUITING | NCT02716246 |
| Adjuvant Immunomodulatory (IM) Therapy | Drug | Phase PHASE3 | Mucopolysaccharidosis IIIA | ENROLLING_BY_INVITATION | NCT04360265 |
| No Investigational Product | Other | Phase PHASE3 | Mucopolysaccharidosis IIIA | ENROLLING_BY_INVITATION | NCT04360265 |
| GTX-102 | Other | Phase PHASE2 | Angelman Syndrome | RECRUITING | NCT07157254 |
| No intervention | Other | Phase PHASE2 | Angelman Syndrome | RECRUITING | NCT07157254 |
| Adjuvant IM Therapy | Drug | Phase PHASE2 | MPS IIIA | RECRUITING | NCT02716246 |
| Optimized Prophylactic IM Therapy | Drug | Phase PHASE2 | MPS IIIA | RECRUITING | NCT02716246 |
| Prophylactic Immunomodulatory (IM) Therapy | Drug | Phase PHASE2 | MPS IIIA | RECRUITING | NCT02716246 |
| UX111 | Other | Phase PHASE2 | MPS IIIA | RECRUITING | NCT02716246 |
| Adjuvant Immunomodulatory (IM) Therapy | Drug | Phase PHASE3 | Mucopolysaccharidosis IIIA | ENROLLING_BY_INVITATION | NCT04360265 |
| No Investigational Product | Other | Phase PHASE3 | Mucopolysaccharidosis IIIA | ENROLLING_BY_INVITATION | NCT04360265 |
| GTX-102 | Other | Phase PHASE2 | Angelman Syndrome | RECRUITING | NCT07157254 |
| No intervention | Other | Phase PHASE2 | Angelman Syndrome | RECRUITING | NCT07157254 |
| Adjuvant IM Therapy | Drug | Phase PHASE2 | MPS IIIA | RECRUITING | NCT02716246 |
| Optimized Prophylactic IM Therapy | Drug | Phase PHASE2 | MPS IIIA | RECRUITING | NCT02716246 |
| Prophylactic Immunomodulatory (IM) Therapy | Drug | Phase PHASE2 | MPS IIIA | RECRUITING | NCT02716246 |
| UX111 | Other | Phase PHASE2 | MPS IIIA | RECRUITING | NCT02716246 |
| Adjuvant Immunomodulatory (IM) Therapy | Drug | Phase PHASE3 | Mucopolysaccharidosis IIIA | ENROLLING_BY_INVITATION | NCT04360265 |
| No Investigational Product | Other | Phase PHASE3 | Mucopolysaccharidosis IIIA | ENROLLING_BY_INVITATION | NCT04360265 |
| GTX-102 | Other | Phase PHASE2 | Angelman Syndrome | RECRUITING | NCT07157254 |
| No intervention | Other | Phase PHASE2 | Angelman Syndrome | RECRUITING | NCT07157254 |
| Adjuvant IM Therapy | Drug | Phase PHASE2 | MPS IIIA | RECRUITING | NCT02716246 |
| Optimized Prophylactic IM Therapy | Drug | Phase PHASE2 | MPS IIIA | RECRUITING | NCT02716246 |
| Prophylactic Immunomodulatory (IM) Therapy | Drug | Phase PHASE2 | MPS IIIA | RECRUITING | NCT02716246 |
| UX111 | Other | Phase PHASE2 | MPS IIIA | RECRUITING | NCT02716246 |
| Adjuvant Immunomodulatory (IM) Therapy | Drug | Phase PHASE3 | Mucopolysaccharidosis IIIA | ENROLLING_BY_INVITATION | NCT04360265 |
| No Investigational Product | Other | Phase PHASE3 | Mucopolysaccharidosis IIIA | ENROLLING_BY_INVITATION | NCT04360265 |
| GTX-102 | Other | Phase PHASE2 | Angelman Syndrome | RECRUITING | NCT07157254 |
| No intervention | Other | Phase PHASE2 | Angelman Syndrome | RECRUITING | NCT07157254 |
| Adjuvant IM Therapy | Drug | Phase PHASE2 | MPS IIIA | RECRUITING | NCT02716246 |
| Optimized Prophylactic IM Therapy | Drug | Phase PHASE2 | MPS IIIA | RECRUITING | NCT02716246 |
| Prophylactic Immunomodulatory (IM) Therapy | Drug | Phase PHASE2 | MPS IIIA | RECRUITING | NCT02716246 |
| UX111 | Other | Phase PHASE2 | MPS IIIA | RECRUITING | NCT02716246 |
| Adjuvant Immunomodulatory (IM) Therapy | Drug | Phase PHASE3 | Mucopolysaccharidosis IIIA | ENROLLING_BY_INVITATION | NCT04360265 |
| No Investigational Product | Other | Phase PHASE3 | Mucopolysaccharidosis IIIA | ENROLLING_BY_INVITATION | NCT04360265 |
| GTX-102 | Other | Phase PHASE2 | Angelman Syndrome | RECRUITING | NCT07157254 |
| No intervention | Other | Phase PHASE2 | Angelman Syndrome | RECRUITING | NCT07157254 |
| Adjuvant IM Therapy | Drug | Phase PHASE2 | MPS IIIA | RECRUITING | NCT02716246 |
| Optimized Prophylactic IM Therapy | Drug | Phase PHASE2 | MPS IIIA | RECRUITING | NCT02716246 |
| Prophylactic Immunomodulatory (IM) Therapy | Drug | Phase PHASE2 | MPS IIIA | RECRUITING | NCT02716246 |
| UX111 | Other | Phase PHASE2 | MPS IIIA | RECRUITING | NCT02716246 |
| Adjuvant Immunomodulatory (IM) Therapy | Drug | Phase PHASE3 | Mucopolysaccharidosis IIIA | ENROLLING_BY_INVITATION | NCT04360265 |
| No Investigational Product | Other | Phase PHASE3 | Mucopolysaccharidosis IIIA | ENROLLING_BY_INVITATION | NCT04360265 |
| GTX-102 | Other | Phase PHASE2 | Angelman Syndrome | RECRUITING | NCT07157254 |
| No intervention | Other | Phase PHASE2 | Angelman Syndrome | RECRUITING | NCT07157254 |
| Adjuvant IM Therapy | Drug | Phase PHASE2 | MPS IIIA | RECRUITING | NCT02716246 |
| Optimized Prophylactic IM Therapy | Drug | Phase PHASE2 | MPS IIIA | RECRUITING | NCT02716246 |
| Prophylactic Immunomodulatory (IM) Therapy | Drug | Phase PHASE2 | MPS IIIA | RECRUITING | NCT02716246 |
| UX111 | Other | Phase PHASE2 | MPS IIIA | RECRUITING | NCT02716246 |
| Adjuvant Immunomodulatory (IM) Therapy | Drug | Phase PHASE3 | Mucopolysaccharidosis IIIA | ENROLLING_BY_INVITATION | NCT04360265 |
| No Investigational Product | Other | Phase PHASE3 | Mucopolysaccharidosis IIIA | ENROLLING_BY_INVITATION | NCT04360265 |
| GTX-102 | Other | Phase PHASE2 | Angelman Syndrome | RECRUITING | NCT07157254 |
| No intervention | Other | Phase PHASE2 | Angelman Syndrome | RECRUITING | NCT07157254 |
| Adjuvant IM Therapy | Drug | Phase PHASE2 | MPS IIIA | RECRUITING | NCT02716246 |
| Optimized Prophylactic IM Therapy | Drug | Phase PHASE2 | MPS IIIA | RECRUITING | NCT02716246 |
| Prophylactic Immunomodulatory (IM) Therapy | Drug | Phase PHASE2 | MPS IIIA | RECRUITING | NCT02716246 |
| UX111 | Other | Phase PHASE2 | MPS IIIA | RECRUITING | NCT02716246 |
| Adjuvant Immunomodulatory (IM) Therapy | Drug | Phase PHASE3 | Mucopolysaccharidosis IIIA | ENROLLING_BY_INVITATION | NCT04360265 |
| No Investigational Product | Other | Phase PHASE3 | Mucopolysaccharidosis IIIA | ENROLLING_BY_INVITATION | NCT04360265 |
| GTX-102 | Other | Phase PHASE2 | Angelman Syndrome | RECRUITING | NCT07157254 |
| No intervention | Other | Phase PHASE2 | Angelman Syndrome | RECRUITING | NCT07157254 |
| Adjuvant IM Therapy | Drug | Phase PHASE2 | MPS IIIA | RECRUITING | NCT02716246 |
| Optimized Prophylactic IM Therapy | Drug | Phase PHASE2 | MPS IIIA | RECRUITING | NCT02716246 |
| Prophylactic Immunomodulatory (IM) Therapy | Drug | Phase PHASE2 | MPS IIIA | RECRUITING | NCT02716246 |
| UX111 | Other | Phase PHASE2 | MPS IIIA | RECRUITING | NCT02716246 |
| Adjuvant Immunomodulatory (IM) Therapy | Drug | Phase PHASE3 | Mucopolysaccharidosis IIIA | ENROLLING_BY_INVITATION | NCT04360265 |
| No Investigational Product | Other | Phase PHASE3 | Mucopolysaccharidosis IIIA | ENROLLING_BY_INVITATION | NCT04360265 |
| GTX-102 | Other | Phase PHASE2 | Angelman Syndrome | RECRUITING | NCT07157254 |
| No intervention | Other | Phase PHASE2 | Angelman Syndrome | RECRUITING | NCT07157254 |
| Adjuvant IM Therapy | Drug | Phase PHASE2 | MPS IIIA | RECRUITING | NCT02716246 |
| Optimized Prophylactic IM Therapy | Drug | Phase PHASE2 | MPS IIIA | RECRUITING | NCT02716246 |
| Prophylactic Immunomodulatory (IM) Therapy | Drug | Phase PHASE2 | MPS IIIA | RECRUITING | NCT02716246 |
| UX111 | Other | Phase PHASE2 | MPS IIIA | RECRUITING | NCT02716246 |
| Adjuvant Immunomodulatory (IM) Therapy | Drug | Phase PHASE3 | Mucopolysaccharidosis IIIA | ENROLLING_BY_INVITATION | NCT04360265 |
| No Investigational Product | Other | Phase PHASE3 | Mucopolysaccharidosis IIIA | ENROLLING_BY_INVITATION | NCT04360265 |
| No Intervention | Other | Preclinical | Glycogen Storage Disease Type Ia | RECRUITING | NCT06636383 |
| GTX-102 | Other | Phase PHASE2 | Angelman Syndrome | RECRUITING | NCT07157254 |
| No intervention | Other | Phase PHASE2 | Angelman Syndrome | RECRUITING | NCT07157254 |
| Adjuvant IM Therapy | Drug | Phase PHASE2 | MPS IIIA | RECRUITING | NCT02716246 |
| Optimized Prophylactic IM Therapy | Drug | Phase PHASE2 | MPS IIIA | RECRUITING | NCT02716246 |
| Prophylactic Immunomodulatory (IM) Therapy | Drug | Phase PHASE2 | MPS IIIA | RECRUITING | NCT02716246 |
| UX111 | Other | Phase PHASE2 | MPS IIIA | RECRUITING | NCT02716246 |
| Adjuvant Immunomodulatory (IM) Therapy | Drug | Phase PHASE3 | Mucopolysaccharidosis IIIA | ENROLLING_BY_INVITATION | NCT04360265 |
| No Investigational Product | Other | Phase PHASE3 | Mucopolysaccharidosis IIIA | ENROLLING_BY_INVITATION | NCT04360265 |
| No Intervention | Other | Preclinical | Glycogen Storage Disease Type Ia | RECRUITING | NCT06636383 |
| GTX-102 | Other | Phase PHASE2 | Angelman Syndrome | RECRUITING | NCT07157254 |
| No intervention | Other | Phase PHASE2 | Angelman Syndrome | RECRUITING | NCT07157254 |
| Adjuvant IM Therapy | Drug | Phase PHASE2 | MPS IIIA | RECRUITING | NCT02716246 |
| Optimized Prophylactic IM Therapy | Drug | Phase PHASE2 | MPS IIIA | RECRUITING | NCT02716246 |
| Prophylactic Immunomodulatory (IM) Therapy | Drug | Phase PHASE2 | MPS IIIA | RECRUITING | NCT02716246 |
| UX111 | Other | Phase PHASE2 | MPS IIIA | RECRUITING | NCT02716246 |
| Adjuvant Immunomodulatory (IM) Therapy | Drug | Phase PHASE3 | Mucopolysaccharidosis IIIA | ENROLLING_BY_INVITATION | NCT04360265 |
| No Investigational Product | Other | Phase PHASE3 | Mucopolysaccharidosis IIIA | ENROLLING_BY_INVITATION | NCT04360265 |
| No Intervention | Other | Preclinical | Glycogen Storage Disease Type Ia | RECRUITING | NCT06636383 |
| No Intervention | Other | Preclinical | Glycogen Storage Disease Type Ia | RECRUITING | NCT06636383 |
| No Intervention | Other | Preclinical | Glycogen Storage Disease Type Ia | RECRUITING | NCT06636383 |
| No Intervention | Other | Preclinical | Glycogen Storage Disease Type Ia | RECRUITING | NCT06636383 |
| No Intervention | Other | Preclinical | Glycogen Storage Disease Type Ia | RECRUITING | NCT06636383 |
| No Intervention | Other | Preclinical | Glycogen Storage Disease Type Ia | RECRUITING | NCT06636383 |
| No Intervention | Other | Preclinical | Glycogen Storage Disease Type Ia | RECRUITING | NCT06636383 |
| No Intervention | Other | Preclinical | Glycogen Storage Disease Type Ia | RECRUITING | NCT06636383 |
| No Intervention | Other | Preclinical | Glycogen Storage Disease Type Ia | RECRUITING | NCT06636383 |
| Triheptanoin | Other | Phase PHASE2 | Medium-chain Acyl-CoA Dehydrogenase Deficiency | SUSPENDED | NCT06067802 |
| Triheptanoin | Other | Phase PHASE2 | Medium-chain Acyl-CoA Dehydrogenase Deficiency | SUSPENDED | NCT06067802 |
| Triheptanoin | Other | Phase PHASE2 | Medium-chain Acyl-CoA Dehydrogenase Deficiency | SUSPENDED | NCT06067802 |
| Triheptanoin | Other | Phase PHASE2 | Medium-chain Acyl-CoA Dehydrogenase Deficiency | SUSPENDED | NCT06067802 |
| Triheptanoin | Other | Phase PHASE2 | Medium-chain Acyl-CoA Dehydrogenase Deficiency | SUSPENDED | NCT06067802 |
| Triheptanoin | Other | Phase PHASE2 | Medium-chain Acyl-CoA Dehydrogenase Deficiency | SUSPENDED | NCT06067802 |
| Triheptanoin | Other | Phase PHASE2 | Medium-chain Acyl-CoA Dehydrogenase Deficiency | SUSPENDED | NCT06067802 |
| Triheptanoin | Other | Phase PHASE2 | Medium-chain Acyl-CoA Dehydrogenase Deficiency | SUSPENDED | NCT06067802 |
| UX016 | DRUG | Phase PHASE1 | GNE Myopathy | NOT_YET_RECRUITING | NCT07511556 |
| Sham-LP | PROCEDURE | Phase PHASE3 | Angelman Syndrome | ACTIVE_NOT_RECRUITING | NCT06617429 |
| Bisphosphonate | DRUG | Phase PHASE3 | Osteogenesis Imperfecta | ACTIVE_NOT_RECRUITING | NCT05768854 |
| Evinacumab | DRUG | Phase PHASE3 | Homozygous Familial Hypercholesterolemia | COMPLETED | NCT05611528 |
| Sodium Acetate | DRUG | Phase PHASE3 | OTC Deficiency | ACTIVE_NOT_RECRUITING | NCT05345171 |
| Placebo for oral corticosteroids | DRUG | Phase PHASE3 | OTC Deficiency | ACTIVE_NOT_RECRUITING | NCT05345171 |
| Oral Corticosteroids | DRUG | Phase PHASE3 | OTC Deficiency | ACTIVE_NOT_RECRUITING | NCT05345171 |
| DTX301 | GENETIC | Phase PHASE3 | OTC Deficiency | ACTIVE_NOT_RECRUITING | NCT05345171 |
| Placebo for oral prednisolone | DRUG | Phase PHASE3 | Glycogen Storage Disease Type IA | COMPLETED | NCT05139316 |
| Oral prednisolone | DRUG | Phase PHASE3 | Glycogen Storage Disease Type IA | COMPLETED | NCT05139316 |
| Setrusumab | BIOLOGICAL | Phase PHASE3 | Osteogenesis Imperfecta | ACTIVE_NOT_RECRUITING | NCT05768854 |
| Venipuncture | PROCEDURE | Preclinical | Mucopolysaccharidoses | ACTIVE_NOT_RECRUITING | NCT05063435 |
| H1 Blocker | DRUG | Phase PHASE1 | Glycogen Storage Disease Type III | TERMINATED | NCT04990388 |
| H2 Blocker | DRUG | Phase PHASE1 | Glycogen Storage Disease Type III | TERMINATED | NCT04990388 |
| Antipyretic | DRUG | Phase PHASE1 | Glycogen Storage Disease Type III | TERMINATED | NCT04990388 |
| UX053 | BIOLOGICAL | Phase PHASE1 | Glycogen Storage Disease Type III | TERMINATED | NCT04990388 |
| Standard of Care (SOC) | DRUG | Phase PHASE1 | Wilson Disease | ACTIVE_NOT_RECRUITING | NCT04884815 |
| UX701 | GENETIC | Phase PHASE1 | Wilson Disease | ACTIVE_NOT_RECRUITING | NCT04884815 |
| Adjuvant Immunomodulatory (IM) Therapy | DRUG | Phase PHASE3 | Mucopolysaccharidosis IIIA | ENROLLING_BY_INVITATION | NCT04360265 |
| No Investigational Product | OTHER | Phase PHASE3 | Mucopolysaccharidosis IIIA | ENROLLING_BY_INVITATION | NCT04360265 |
| Crysvita (burosumab-twza) Treatment | DRUG | Phase PHASE4 | Epidermal Nevus Syndrome | COMPLETED | NCT04320316 |
| [6,6-2H2]glucose | BIOLOGICAL | Phase PHASE1 | Glycogen Storage Disease Type IA | COMPLETED | NCT04311307 |
| GTX-102 | DRUG | Phase PHASE2 | Angelman Syndrome | RECRUITING | NCT07157254 |
| ABO-102 | DRUG | Phase PHASE1 | MPS IIIA | TERMINATED | NCT04088734 |
| Mepsevii | DRUG | Preclinical | MPS VII | AVAILABLE | NCT03775174 |
| No intervention | OTHER | Phase PHASE2 | Angelman Syndrome | RECRUITING | NCT07157254 |
| Placebo Oil | DRUG | Phase PHASE2 | Tarui Disease | COMPLETED | NCT03642860 |
| No Intervention | OTHER | Preclinical | Glycogen Storage Disease Type Ia | RECRUITING | NCT06636383 |
| Burosumab | DRUG | Phase EARLY_PHASE1 | Cutaneous Skeletal Hypophosphatemia Syndrome (CSHS) | UNKNOWN | NCT03993821 |
| steroid regimen | DRUG | Phase PHASE1 | GSD1 | COMPLETED | NCT03517085 |
| DTX401 | GENETIC | Phase PHASE3 | Glycogen Storage Disease Type IA | COMPLETED | NCT05139316 |
| zoledronic acid (optional) | DRUG | Phase PHASE2 | Osteogenesis Imperfecta, Type I | COMPLETED | NCT03118570 |
| setrusumab | BIOLOGICAL | Phase PHASE3 | Osteogenesis Imperfecta | ACTIVE_NOT_RECRUITING | NCT06636071 |
| Tridecanoic Acid | DRUG | Phase PHASE2 | Rett Syndrome | UNKNOWN | NCT03059160 |
| Prophylactic Corticosteroid Taper Regimen | DRUG | Phase PHASE1 | Ornithine Transcarbamylase (OTC) Deficiency | COMPLETED | NCT02991144 |
| Reactive Corticosteroid Taper Regimen | DRUG | Phase PHASE1 | Ornithine Transcarbamylase (OTC) Deficiency | COMPLETED | NCT02991144 |
| scAAV8OTC | GENETIC | Phase PHASE1 | Ornithine Transcarbamylase (OTC) Deficiency | COMPLETED | NCT02991144 |
| Aceneuramic Acid Extended-Release Tablets | DRUG | Phase PHASE3 | Hereditary Inclusion Body Myopathy | TERMINATED | NCT02736188 |
| Aceneuramic Acid Extended-Release | DRUG | Phase PHASE2 | Hereditary Inclusion Body Myopathy | TERMINATED | NCT02731690 |
| Adjuvant IM Therapy | DRUG | Phase PHASE2 | MPS IIIA | RECRUITING | NCT02716246 |
| Optimized Prophylactic IM Therapy | DRUG | Phase PHASE2 | MPS IIIA | RECRUITING | NCT02716246 |
| Prophylactic Immunomodulatory (IM) Therapy | DRUG | Phase PHASE2 | MPS IIIA | RECRUITING | NCT02716246 |
| UX111 | BIOLOGICAL | Phase PHASE2 | MPS IIIA | RECRUITING | NCT02716246 |
| POST Triheptanoin | DRUG | Phase PHASE1 | Frontal Lobe Hypometabolism | COMPLETED | NCT02679235 |
| DTX101 | GENETIC | Phase PHASE1 | Hemophilia B | TERMINATED | NCT02618915 |
| Triheptanoin oil | DRUG | Phase PHASE2 | Huntington Disease | COMPLETED | NCT02453061 |
| Placebo oil | OTHER | Phase PHASE2 | Glycogen Storage Disease Type V | COMPLETED | NCT02432768 |
| aceneuramic acid extended-release (Ace-ER) | DRUG | Phase PHASE3 | Hereditary Inclusion Body Myopathy | COMPLETED | NCT02377921 |
| Sialic Acid Extended Release (SA-ER) | DRUG | Phase PHASE2 | GNE Myopathy | COMPLETED | NCT01517880 |
| Sialic Acid Extended Release (SA-ER) Tables | DRUG | Phase PHASE1 | Hereditary Inclusion Body Myopathy (HIBM) | COMPLETED | NCT01359319 |
| Sialic Acid Extended Release (SA-ER) Tablets | DRUG | Phase PHASE1 | Hereditary Inclusion Body Myopathy (HIBM) | COMPLETED | NCT01359319 |
| GLUT1 DS | DRUG | Phase PHASE2 | Glut1 Deficiency Syndrome | COMPLETED | NCT02014883 |
| UX007 | DRUG | Phase PHASE3 | Glucose Transporter Type 1 Deficiency Syndrome (Glut1 DS) | TERMINATED | NCT02960217 |
| UX003 | DRUG | Phase PHASE3 | Sly Syndrome | COMPLETED | NCT02432144 |
| SA-IR 500 mg | DRUG | Phase PHASE2 | GNE Myopathy | COMPLETED | NCT01830972 |
| SA-ER 500 mg | DRUG | Phase PHASE2 | GNE Myopathy | COMPLETED | NCT01830972 |
| Placebo | OTHER | Phase PHASE1 | GNE Myopathy | NOT_YET_RECRUITING | NCT07511556 |
| triheptanoin | DRUG | Phase PHASE2 | Rett Syndrome | UNKNOWN | NCT02696044 |
| BPS804 | DRUG | Phase PHASE2 | Osteogenesis Imperfecta | WITHDRAWN | NCT03216486 |
| Placebo to 20mg/Kg BPS804 | DRUG | Phase PHASE2 | Osteopenia | COMPLETED | NCT01406548 |
| BPS804 20mg/Kg | DRUG | Phase PHASE2 | Osteopenia | COMPLETED | NCT01406548 |
| Vegetable Oil | OTHER | Phase PHASE2 | Adult Polyglucosan Body Disease | COMPLETED | NCT00947960 |
| Triheptanoin | DRUG | Phase PHASE3 | Long-chain Fatty Acid Oxidation Disorders (LC-FAOD) | ACTIVE_NOT_RECRUITING | NCT05933200 |
Total products: 359