Expanded Access to Mepsevii
- Status
- AVAILABLE
- Phase
- N/A
Clinical Trials Related to Ultragenyx Pharmaceutical Inc
Related studies linked through entity extraction aliases.
Expanded Access to Triheptanoin
- Status
- AVAILABLE
- Phase
- N/A
Clinical Outcome of Triheptanoin Treatment in Patients With Long-chain Fatty Acid Oxidation Disorders (LC-FAOD) Treated Under Expanded Access Program
- Status
- COMPLETED
- Phase
- N/A
Anaplerotic Therapy Using Triheptanoin for Patients With Glycogen Storage Disease Type I
- Status
- COMPLETED
- Phase
- EARLY_PHASE1
X-linked Hypophosphatemia Disease Monitoring Program
- Status
- ACTIVE_NOT_RECRUITING
- Phase
- N/A
The Effect of Triheptanoin on Fatty Acid Oxidation and Exercise Tolerance in Patients With Glycogenoses
- Status
- COMPLETED
- Phase
- PHASE2
Long Term Follow Up to Evaluate DTX301 in Adults With Late-Onset OTC Deficiency
- Status
- ACTIVE_NOT_RECRUITING
- Phase
- N/A
Mucopolysaccharidosis VII Disease Monitoring Program
- Status
- RECRUITING
- Phase
- N/A
Open Label Trial Assessing Safety and Efficacy of Burosumab (KRN23), in a Patient With ENS and Hypophosphatemic Rickets
- Status
- COMPLETED
- Phase
- PHASE3
Safety and Dose-Finding Study of DTX401 (AAV8G6PC) in Adults With Glycogen Storage Disease Type Ia (GSDIa)
- Status
- COMPLETED
- Phase
- PHASE1/PHASE2
A Pilot Trial of Triheptanoin for People With Amyotrophic Lateral Sclerosis (PALS)
- Status
- COMPLETED
- Phase
- PHASE1/PHASE2
An Exploratory Study of BPS804 Treatment in Adult Patients With Type I, III or IV Osteogenesis Imperfecta
- Status
- WITHDRAWN
- Phase
- PHASE2
A Study in Adult Patients With Type I, III or IV Osteogenesis Imperfecta Treated With BPS804
- Status
- COMPLETED
- Phase
- PHASE2
Open Label Trial of Triheptanoin (UX007) in Treatment of Rett Syndrome.
- Status
- UNKNOWN
- Phase
- PHASE2
Safety and Dose-Finding Study of DTX301 (scAAV8OTC) in Adults With Late-Onset Ornithine Transcarbamylase (OTC) Deficiency
- Status
- COMPLETED
- Phase
- PHASE1/PHASE2
Long-Term Safety, Tolerability, and Efficacy of DTX101 (AAVrh10FIX) in Adults With Moderate/Severe to Severe Hemophilia B
- Status
- COMPLETED
- Phase
- N/A
Treatment With UX007 for a Single Patient With GLUT1 Deficiency Syndrome
- Status
- NO_LONGER_AVAILABLE
- Phase
- N/A
Crossover Study to Assess the Efficacy and Safety of UX007 in the Treatment of Movement Disorders Associated With Glucose Transporter Type 1 Deficiency Syndrome (Glut1 DS)
- Status
- TERMINATED
- Phase
- PHASE3
Observational Study of Males With Creatine Transporter Deficiency
- Status
- TERMINATED
- Phase
- N/A
Study to Evaluate the Safety and Efficacy of Aceneuramic Acid Extended-Release (Ace-ER) Tablets in Patients With Glucosamine (UDP-N-acetyl)-2-epimerase Myopathy (GNEM) or Hereditary Inclusion Body Myopathy (HIBM)
- Status
- TERMINATED
- Phase
- PHASE3