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 ⌃Compassionate Use Policy
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 Compassionate Use Policy ⌃