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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 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
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
Wave’s challenge to Sarepta in Duchenne falls flat

The demise of Wave’s programmes leaves Japan’s Nippon Shinyaku Pharma as the main rival to Sarepta in DMD. ... DMD is one of the most common fatal genetic disorders, affecting around one in every 3, 500-5, 000 male births worldwide.
FDA grants surprise OK to Sarepta’s DMD therapy after earlier rejection

These patients represent around 8% of the DMD population, while those with the exon 51 mutation account for around 13%. ... benefit to DMD patients awaiting treatment,” said the company’s chief executive Doug Ingram.
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

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Latest Intelligence

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

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

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

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.
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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