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sapropterin dihydrochloride

This page shows the latest sapropterin dihydrochloride news and features for those working in and with pharma, biotech and healthcare.

FDA pauses BioMarin gene therapy trial

FDA pauses BioMarin gene therapy trial

It’s products include Vimizim (elosulfase alfa) for MPS type IVA (Morquio A syndrome), Naglazyme (galsulfase) for MPS VI (Maroteaux-Lamy syndrome) and Kuvan (sapropterin dihydrochloride) in children with PKU.

Latest news

  • Judge forces NHS England rethink on Kuvan Judge forces NHS England rethink on Kuvan

    BioMarin won EU approval in 2008 for Kuvan (sapropterin dihydrochloride), a drug that is used alongside a phenylalanine-restricted diet to reduce levels of amino acid even further, but so far

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Latest Intelligence

  • Deal Watch October 2015 Deal Watch October 2015

    In the original PKU-focused deal dating back to 2005, Merck signed an exclusive licence with BioMarin for the oral drug Kuvan (sapropterin dihydrochloride), which is now marketed, and an option

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