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  2. US FDA approves Italfarmaco's drug for Duchenne muscle ... - AOL

    www.aol.com/news/us-fda-approves-italfarmacos...

    The U.S. FDA has approved privately held Italfarmaco Group's drug to treat Duchenne muscular dystrophy (DMD), an inherited muscle-wasting disorder, the health regulator said on Thursday. The oral ...

  3. Ataluren - Wikipedia

    en.wikipedia.org/wiki/Ataluren

    In May 2014, ataluren received a positive opinion from the Committee for Medicinal Products for Human Use (CHMP) of the European Medicines Agency (EMA) [18] and received market authorization from the European Commission to treat people with nonsense mutation Duchenne muscular dystrophy in August 2014; [2] a confirmatory phase III clinical trial ...

  4. Duchenne muscular dystrophy - Wikipedia

    en.wikipedia.org/wiki/Duchenne_muscular_dystrophy

    Duchenne muscular dystrophy is a rare progressive disease that eventually affects all voluntary muscles and involves the heart and breathing muscles in later stages. Life expectancy is estimated to be around 25–26, [18] [59] but this varies. People born with Duchenne muscular dystrophy after 1990 have a median life expectancy of approximately ...

  5. US FDA approves expanded use of Sarepta's Duchenne gene ... - AOL

    www.aol.com/news/us-fda-approves-expanded...

    (Reuters) -The U.S. Food and Drug Administration allowed the expanded use of Sarepta Therapeutics' gene therapy for patients with Duchenne muscular dystrophy aged four and older on Thursday ...

  6. Givinostat - Wikipedia

    en.wikipedia.org/wiki/Givinostat

    Givinostat is in numerous phase II clinical trials (including for relapsed leukemias and myelomas), [15] and has been granted orphan drug designation in the European Union for the treatment of systemic juvenile idiopathic arthritis, [16] polycythaemia vera. [11] and Duchenne muscular dystrophy.

  7. Santhera's drug gets US FDA nod for rare muscular dystrophy - AOL

    www.aol.com/news/us-fda-approves-santheras-drug...

    (Reuters) -Catalyst Pharmaceuticals said on Thursday that U.S. health regulators have approved its partner Santhera Pharmaceuticals' drug to treat Duchenne muscular dystrophy (DMD) in patients ...

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