Ultragenyx Pharmaceutical Inc

MPS IIIA Disease Monitoring Program

Role: lead

Setrusumab in Pediatric Japanese Subjects With Osteogenesis Imperfecta

Role: lead

A Phase 1/2/3 Study of UX701 Gene Therapy in Adults With Wilson Disease

Role: lead

Phase I/II/III Gene Transfer Clinical Trial of scAAV9.U1a.hSGSH

Role: lead

A Study to Determine the Effect of Triheptanoin Compared With Even-Chain MCT on MCEs in Pediatric Patients With LC-FAOD

Role: lead

A Safety and Efficacy Study of GTX-102 in Subjects With Deletion- or Nondeletion-type Angelman Syndrome (AS)

Role: lead

Long-term Extension of GTX-102 in Angelman Syndrome

Role: lead

Clinical Study of DTX301 AAV-Mediated Gene Transfer for Ornithine Transcarbamylase (OTC) Deficiency

Role: lead

First-in-human Study of UX016 in GNEM

Role: lead

X-linked Hypophosphatemia Disease Monitoring Program

Role: lead

Mucopolysaccharidosis VII Disease Monitoring Program

Role: lead

Glycogen Storage Disease Type Ia (GSDIa) Disease Monitoring Program

Role: lead

Assessing the Impact of Intensification of Lipid Lowering Therapy With Guidelines-based Evinacumab Administration on Coronary Plaque Volumes Measured by Coronary Computed Tomography Angiography (CCTA) in Patients With Homozygous Familial Hypercholesterolemia (HoFH)

Role: collaborator

Phase 3 Efficacy and Safety Study of GTX-102 in Pediatric Subjects With Angelman Syndrome (AS)

Role: lead

Expanded Access to Triheptanoin

Role: lead

Expanded Access to Mepsevii

Role: lead

Burosumab for CSHS

Role: collaborator

ScreenPlus: A Comprehensive, Flexible, Multi-disorder Newborn Screening Program

Role: collaborator

Follow-up Study of AAV-Mediated Gene Transfer (UX111; Previously Known as ABO-102) for MPS Type IIIA

Role: lead

Study to Evaluate the Use of Triheptanoin in Patients With Medium Chain Acyl-CoA Dehydrogenase Deficiency (MCADD)

Role: collaborator