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DMD

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

Lilly links up with Precision BioSciences in genome editing deal

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).

Latest news

  • FDA approves Sarepta rival NS Pharma’s Duchenne drug 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%.

  • FDA sets Q3 decision date for NS Pharma’s DMD drug 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

  • Solid Bio axes staff by one-third as it clings to troubled DMD programme 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 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 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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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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