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DMD

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

CHMP hands Santhera second ‘no’ in Duchenne muscular dystrophy

CHMP hands Santhera second ‘no’ in Duchenne muscular dystrophy

European regulatory advisers have refused to shift their negative position on Santhera’s Duchenne muscular dystrophy (DMD) drug Raxone (idebenone). ... reduces the risk of bronchopulmonary complications and hospitalisation in patients with DMD not

Latest news

  • Transforming access in rare diseases Transforming access in rare diseases

    In Duchenne muscular dystrophy (DMD), for example, treatments from gene therapy to stem cell transplantation are all on the horizon, and all will need to be assessed by country healthcare systems ... In DMD, a standard outcome measure is the six-minute

  • Duchenne UK and pharma partner pilot data-sharing HTA approach Duchenne UK and pharma partner pilot data-sharing HTA approach

    A new initiative between medical charity Duchenne UK and several pharma companies will try to  increase the chances of patients with Duchenne muscular dystrophy (DMD) of accessing innovative treatments. ... The pharma partners - Roche, Sarepta

  • Sanofi launches French eHealth laboratory 39BIS Sanofi launches French eHealth laboratory 39BIS

    Dmd Santé, France eHealth Tech and Umanlife are among the laboratory’s initial external partners and its offering will include a six-month project process.

  • FDA reviewer unimpressed with PTC’s Translarna FDA reviewer unimpressed with PTC’s Translarna

    that the firm was emboldened by the FDA’s approval last year of another DMD therapy from Sarepta - Exondys 51 (eteplirsen) - after Janet Woodcock, head of the agency’s Center for ... saying its commercial operations were already in gear following the

  • Sarepta eyes FDA filing for DMD drug golodirsen after positive trial Sarepta eyes FDA filing for DMD drug golodirsen after positive trial

    price is justified as it has already spent around $1bn developing its DMD therapies. ... Sarepta says its goal is to be able to prove a treatment for 100% of all DMD patients.

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Latest Intelligence

  • 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. ... Prosensa has been busy since drisapersen failed a pivotal phase III for DMD (failure to beat 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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