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DMD
This page shows the latest DMD news and features for those working in and with pharma, biotech and healthcare.
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
Latest news
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Lilly links up with Precision BioSciences in genome editing deal
A number of companies are seeking to develop therapies for DMD as the therapy area becomes increasingly more competitive. ... Vyondys 53 is Sarepta’s second approved DMD therapy after Exondys 51 (eteplirsen).
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FDA approves Sarepta rival NS Pharma’s Duchenne drug
The approval of Viltepso was based on two clinical studies with a total of 32 patients with genetically confirmed DMD. ... This represents around 8% of the overall DMD population, while those with the exon 51 mutation account for around 13%.
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FDA sets Q3 decision date for NS Pharma’s DMD drug
DMD causes progressive weakness and atrophy of skeletal, heart and pulmonary muscles, and overwhelmingly affects males. ... NS Pharma’s drug is designed to treat DMD patients with exon 53 mutations – the exact same mutation which rival Sarepta’s
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Solid Bio axes staff by one-third as it clings to troubled DMD programme
Extends company's cash runway up until 2021. Solid Biosciences has sacrificed a third of its staff as it desperately tries to cling to its troubled Duchenne muscular dystrophy (DMD) programme. ... The FDA halted the IGNITE DMD trial following a report of
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Roche pays $1.15bn for Sarepta’s Duchenne gene therapy
DMD – a rare muscle-wasting disease that mostly affects boys and typically is fatal by age 30 – is caused by a defect in the gene coding for dystrophin, a protein found ... functionality of the mutated protein and avoiding the contraction-induced
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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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Latest appointments
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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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