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DMD

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

FDA rejects Sarepta’s new Duchenne drug over safety fears

FDA rejects Sarepta’s new Duchenne drug over safety fears

These patients represent around 8% of the DMD population, while those with the exon 51 mutation account for around 13%. ... The company is also investing in gene therapies for DMD and a range of other rare diseases as well as multiple sclerosis.

Latest news

  • Sarepta investigating mistakenly submitted Duchenne therapy report Sarepta investigating mistakenly submitted Duchenne therapy report

    rhabdomyolysis. This is a serious condition which is a commonly understood risk associated with DMD and can lead to complications such as kidney failure. ... Sarepta has been the lead in the DMD market, with some studies of its approved gene therapy

  • Rivals rise as safety issue mars Pfizer’s DMD gene therapy trial Rivals rise as safety issue mars Pfizer’s DMD gene therapy trial

    Company to press ahead with phase 3 trial. Data from a trial of Pfizer’s gene therapy for Duchenne muscular dystrophy (DMD) have shown signs of efficacy, but also safety concerns ... Typically children with DMD would have stable NSAA scores or see a

  • Vertex eyes Duchenne market with Exonics, CRISPR deals Vertex eyes Duchenne market with Exonics, CRISPR deals

    First up is a deal to acquire US biotech Exonics Therapeutics, which is developing gene-editing therapies for DMD and other genetic neuromuscular diseases including myotonic dystrophy type 1 (DM1). ... That deal also focuses on gene-editing technology

  • Ex-Pharmacyclics CEO invests $25m in Summit Ex-Pharmacyclics CEO invests $25m in Summit

    It’s also a boost to the UK company after the disappointment of its recent failed programme in Duchenne muscular dystrophy (DMD), which prompted its re-focusing as an anti-infectives

  • Entering the era of digital trials Entering the era of digital trials

    A version of the phenomenon affects trials in muscle disorders such as Duchenne muscular dystrophy (DMD). ... One study found kids with DMD walked 11% further if they were offered $50 (£39) to beat their previous distance.

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Latest Intelligence

  • 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. ... $20.31 before settling at around $19.00.  Prosensa has been busy since drisapersen failed a

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Latest appointments

  • 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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Latest from PMHub

  • 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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