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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 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%.
Latest news
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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
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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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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.
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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.
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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. ... $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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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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COVID-19 Updates and Daily News
Featured jobs
- New study identifies drugs that are safe to use in COVID-19 treatment
- Gavi says 76 wealthy countries have committed to COVAX vaccine facility
- Gilead agrees $21bn buyout of Immunomedics
- 20 for 2020 – Five pharma companies to watch
- FDA approves Roche’s Actemra COVID-19 trial
- Regeneron launches phase 3 COVID-19 antibody trial in the UK
- FDA hands BMS and bluebird bio a priority review for ide-cel
- Janssen’s EGFR-targeting treatment regimen shows early benefit in lung cancer
- Relief Therapeutics seeks US emergency approval for COVID-19 drug
- Could formulary intelligence be your brand differentiator?
- How COVID-19 is accelerating the threat of antimicrobial resistance
- Porterhouse Medical strengthens its senior management team
PMHub
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REACH, ENGAGE & MEASURE - Accomplish meaningful HCP engagement via Medthority, our trusted independent medical website. Support education and treatment...
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- Could formulary intelligence be your brand differentiator?
- Proprietary research, and the COVID-19 experience, shows how formulary intelligence can drive competitive advantage at launch...
- How COVID-19 is accelerating the threat of antimicrobial resistance
- Why antimicrobial resistance needs to be addressed with the same urgency as COVID-19...
- Improve clinical trials through patient journey mapping
- Could patient journey mapping be your solution to improving clinical study recruitment and retention? While awareness of clinical studies is low across the population, it’s not the only issue. It’s...
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