Ultragenyx Pharmaceutical, Inc
Ultragenyx is dedicated to advancing innovative medicines for rare and ultrarare diseases, with a focus on creating transformative therapies and supporting communities affected by these conditions. The company has a robust pipeline of clinical programs and a mission to do the right things for patients during development and commercialization. It also invests in cutting-edge gene therapy manufacturing facilities and collaborates with patient communities to accelerate research and development of treatments.
Industries
Nr. of Employees
large (251-1000)
Ultragenyx Pharmaceutical, Inc
Novato, California, United States, North America
Products
CRYSVITA
A therapy for X-linked hypophosphatemia (XLH), a rare genetic disorder that affects bone mineralization.
MEPSEVII
An enzyme replacement therapy for mucopolysaccharidosis VII (MPS VII), a rare lysosomal storage disorder.
DOJOLVI
A triheptanoin-based therapy for long-chain fatty acid oxidation disorders (LC-FAOD), which are metabolic conditions affecting energy production.
UX143 (setrusumab)
An investigational monoclonal antibody for osteogenesis imperfecta (OI), aimed at improving bone formation and density.
GTX-102
An investigational antisense oligonucleotide therapy for Angelman Syndrome (AS), designed to reactivate the UBE3A gene.
UltraCare
A patient support program providing assistance with insurance coverage, financial support, and access to resources for rare disease treatments.
CRYSVITA
A therapy for X-linked hypophosphatemia (XLH), a rare genetic disorder that affects bone mineralization.
MEPSEVII
An enzyme replacement therapy for mucopolysaccharidosis VII (MPS VII), a rare lysosomal storage disorder.
DOJOLVI
A triheptanoin-based therapy for long-chain fatty acid oxidation disorders (LC-FAOD), which are metabolic conditions affecting energy production.
UX143 (setrusumab)
An investigational monoclonal antibody for osteogenesis imperfecta (OI), aimed at improving bone formation and density.
GTX-102
An investigational antisense oligonucleotide therapy for Angelman Syndrome (AS), designed to reactivate the UBE3A gene.
UltraCare
A patient support program providing assistance with insurance coverage, financial support, and access to resources for rare disease treatments.