Available Products
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ROCTAVIAN™ (valoctocogene roxaparvovec-rvox)
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VOXZOGO® (vosoritide)
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PALYNZIQ® (pegvaliase-pqpz) Injection
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BRINEURA® (cerliponase alfa)
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VIMIZIM® (elosulfase alfa)
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KUVAN® (sapropterin dihydrochloride) Tablets for Oral Use and Powder for Oral Solution
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NAGLAZYME® (galsulfase)
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ALDURAZYME® (laronidase)
Our Pipeline
We maximize the impact of genetic innovation to develop transformative therapies.
Each drug candidate we pursue is guided by a fundamental understanding of the genetics and underlying biology of the condition it will address.
Learn More about our pipeline ⌃Clinical Trials
Our thoughtful approach to clinical trial design enables us to move fast and efficiently while protecting the safety of our patients.
Learn More about our clinical trials ⌃Expanded Access
We are committed to developing safe and effective therapies and providing those therapies to the broadest group of patients as quickly as possible.
Learn More Expanded Access ⌃