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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
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.
Latest news
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FDA advisory committee backs Sarepta’s Duchenne muscular dystrophy gene therapy
A US Food and Drug Administration’s (FDA) panel of experts has recommended Sarepta Therapeutics’ Duchenne muscular dystrophy (DMD) investigational gene therapy for accelerated approval. ... SRP-9001 is designed to address the underlying cause of DMD
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Sarepta to assess imlifidase as pre-treatment to Duchenne muscular dystrophy gene therapy
AAV-based gene therapy for the treatment of Duchenne muscular dystrophy (DMD), the company’s partner Hansa Biopharma AB (Hansa) announced. ... Sarepta submitted a Biologics License Application to the US Food and Drug Administration in September for the
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FDA receives Santhera and ReveraGen’s application for Duchenne muscular dystrophy treatment
DMD is a rare inherited X-chromosome-linked disease, almost exclusively affecting males, and is caused by a change or mutation in the gene that encodes instructions for dystrophin, meaning those ... as Promising Innovative Medicine status from the UK
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Sarepta to submit application for Duchenne muscular dystrophy gene therapy
DMD). The announcement follows updated clinical trial results from the company’s ENDEAVOR study in July, which bolstered the data for SRP-9001 for efficacy and durability. ... DMD affects approximately one in every 3, 500-5, 000 newborn males worldwide.
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Sarepta Therapeutics’ DMD gene therapy shows early success
Sarepta Therapeutics has revealed some early promising data for its Duchenne muscular dystrophy (DMD), although the company also reported serious adverse events associated with the treatment. ... Roche. The study, which has so far enrolled 20
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Latest Intelligence
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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.
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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
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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Latest from PMHub
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Delivering change through better rare disease communications
Benjamin is a trustee and patient advocate for DMD Pathfinders and has recently completed a Masters in Scientific Communications.
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Solaris Health looks at the gene therapy revolution
for instance to treat haemophilia, rare ophthalmic disorders and for Duchenne muscular dystrophy/DMD), genetic therapies are likely to represent a revolution in medicine over the coming years. ... However, in most areas where genetic treatments are being
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