Ultragenyx Pharmaceutical Inc

Setrusumab in Pediatric Japanese Subjects With Osteogenesis Imperfecta

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

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

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)

Triheptanoin for Children With Primary-Specific Pyruvate Dehydrogenase Complex (PDC) Deficiency

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

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

Long Term Follow Up to Evaluate DTX301 in Adults With Late-Onset OTC Deficiency

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

Tumor-induced Osteomalacia Disease Monitoring Program

Mucopolysaccharidosis VII Disease Monitoring Program

X-linked Hypophosphatemia Disease Monitoring Program

Long-Chain Fatty Acid Oxidation Disorders In-Clinic Disease Monitoring Program

Glycogen Storage Disease Type Ia (GSDIa) Disease Monitoring Program