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muscular dystrophy

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

Vertex eyes Duchenne market with Exonics, CRISPR deals

Vertex eyes Duchenne market with Exonics, CRISPR deals

Biotech has gene-editing expertise. Vertex Pharma has agreed a pair of deals to build a position in gene-editing therapeutics for diseases like Duchenne muscular dystrophy. ... DM1 is the most common adult muscular dystrophy, and is also caused by

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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 October 2016 Deal Watch October 2016

    767. Sarepta (US). Summit (UK). Licence collaboration. Europe Utrophin modulator pipeline, lead molecule phase II for 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

    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 CAT-1004 for Duchenne muscular dystrophy (DMD), and our lipid

  • 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, David will make a major contribution to

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Anatomy Health

We are a health information design consultancy focused on developing simple, accessible and inclusive patient communications. From evaluating existing patient...

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