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Duchenne muscular dystrophy

This page shows the latest Duchenne muscular dystrophy 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

CHMP hands Santhera second ‘ no’ in Duchenne muscular dystrophy. Swiss biopharma says it will re-file Raxone for the licence extension. ... European regulatory advisers have refused to shift their negative position on Santhera’s Duchenne muscular

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 ... Phase one of HERCULES is working to provide a model of

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

    Duchenne UK and pharma partner pilot data-sharing HTA approach. Pfizer, Roche and PTC Therapeutics are among those participating in Project Hercules. ... A new initiative between medical charity Duchenne UK and several pharma companies will try to 

  • Alexion's Soliris gets US approval for myasthenia gravis Alexion's Soliris gets US approval for myasthenia gravis

    according to Muscular Dystrophy Association (MDA). ... s programme - including periodic paralysis, Duchenne muscular dystrophy, amyotrophic lateral sclerosis, spinal muscular atrophy and now MG”.

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

    PTC Therapeutics’Duchenne muscular dystrophy drug Translarna is up for an FDA advisory committee meeting on Thursday - but is facing resistance from the agency’s reviewer. ... PTC recently shared additional data from a phase III trial of Translarna

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

    The exon-skipping drug raised functional dystrophin levels. Sarepta is preparing to file a follow-up to its Duchenne muscular dystrophy (DMD) treatment Exondys 51 that seems to have greater efficacy.

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Latest Intelligence

  • Europe vs the US: New drug product approvals Europe vs the US: New drug product approvals

    Among the noteworthy orphan approvals are Exondys 51 (for the treatment of Duchenne muscular dystrophy, only approved in the US), Darzalex (for the treatment of multiple myeloma, approved in the US ... 2015 and in the EU 2016) and Spinraza (for the

  • Deal Watch - May 2017 Deal Watch - May 2017

    397. BMS (US). Roche (CH). Licence. Anti-myostatin adnectin pre phase 2 for Duchenne Muscular Dystrophy .

  • Deal Watch March 2017 Deal Watch March 2017

    The other asset purchase agreement was made by PTC Therapeutics, who acquired Emflaza, a steroid approved for Duchenne Muscular Dystrophy, from Marathon Pharmaceuticals for $140m upfront ($75m in cash and $65m ... 230. Marathon. PTC Therapeutics. Product

  • Deal Watch February 2017 Deal Watch February 2017

    Licence. 145. Sarepta Therapeutics/ Gilead Sciences. PRV received when EXONDYS 51 was FDA approved for Duchenne muscular dystrophy.

  • Deal Watch table for November 2014 Deal Watch table for November 2014

    Licence. 935. Prosensa / BioMarin. Drisapersen – for Duchenne muscular dystrophy.

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Latest appointments

  • Catabasis hires Rick Modi as its chief business officer Catabasis hires Rick Modi as its chief business officer

    Catabasis hires Rick Modi as its chief business officer. Will help the firm develop its flagship Duchenne muscular dystrophy product. ... We look forward to leveraging his capabilities as we advance our clinical development pipeline, particularly

  • Summit appoints Pfizer/Bayer veteran as CMO Summit appoints Pfizer/Bayer veteran as CMO

    Summit appoints Pfizer/Bayer veteran as CMO. David Roblin to lead development of therapies for Duchenne muscular dystrophy and C.difficile. ... As our Duchenne muscular dystrophy and C difficile infection drug programmes go through patient clinical trials

More from appointments
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More from PMHub
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