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DMD

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

Decision date for Sarepta’s Duchenne muscular dystrophy gene therapy delayed by FDA

Decision date for Sarepta’s Duchenne muscular dystrophy gene therapy delayed by FDA

DMD aged four to five years on 22 June this year, rather than the originally proposed 29 May date. ... SRP-9001 is designed to address the underlying cause of DMD through the targeted production of functional components of dystrophin in muscle tissue.

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  • Tackling a formidable foe Tackling a formidable foe

    Like other DMDs, metal exposure should be avoided,” adds Ben- Amor, who has been involved in MS research and business operations for 20 years.

  • Pharma deals during November 2014 Pharma deals during November 2014

    additional $160m more in near-term milestones if drisapersen, the late-stage drug for Duchenne muscular dystrophy (DMD), secures “early” approval. ... Prosensa has been busy since drisapersen failed a pivotal phase III for DMD (failure to beat a

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  • Catabasis hires Rick Modi as its chief business officer Catabasis hires Rick Modi as its chief business officer

    We look forward to leveraging his capabilities as we advance our clinical development pipeline, particularly CAT-1004 for Duchenne muscular dystrophy (DMD), and our lipid programmes, CAT-2003 and CAT-2054.”. ... we seek to bring disease-modifying

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