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DMD
This page shows the latest DMD news and features for those working in and with pharma, biotech and healthcare.
Solid Bio falls as FDA halts DMD trial over safety concerns
The FDA has halted the IGNITE DMD trial because of safety concerns, after a patient experienced a serious adverse event (SAE) which was deemed related to the drug. ... In the meantime, rival candidate viltolarsen from Japanese pharma Nippon Shinyaku (NS
Latest news
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Sarepta bounces back with new gene therapy data
Announces positive results in treatment of limb-girdle muscular dystrophy. Following the disappointing FDA rejection of its latest treatment for Duchenne Muscular Dystrophy (DMD) in August, Sarepta Therapeutics has scored positive
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Sarepta rival NS Pharma files Duchenne drug with FDA
Sarepta was dealt a significant blow in August when its new DMD treatment Vyondys 53 was rejected by the FDA. ... These patients represent around 8% of the DMD population, while those with the exon 51 mutation account for around 13%.
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Vertex eyes type 1 diabetes cure with biotech acquisition
Exonics is developing gene-editing therapies for DMD and other genetic neuromuscular disease. ... That deal also focuses on gene-editing technology directed at DMD and DM1.
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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.
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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
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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. ... $20.31 before settling at around $19.00. Prosensa has been busy since drisapersen failed 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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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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