Fast track for Muscular Dystrophy treatment

The US FDA has given fast track approval to Israel’s BioBlast for its Cabaletta treatment for two rare and currently untreatable conditions – oculopharyngeal muscular dystrophy (OPMD) and spinocerebellar ataxia type 3 (SCA3).

http://www.israel21c.org/drug-for-rare-muscular-dystrophy-fast-tracked/

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2 Responses to Fast track for Muscular Dystrophy treatment

  1. Aizhan says:

    Good day!
    I’m from Kazakhstan. Two my nephews have Duchenne Muscular Dystrophy. They are 6 and 8 years old. Can I get any kinds of tratment or medicine?
    Thank you!

    • MichaelOrdman says:

      Hi Aizhan.

      I just report on Israeli treatments which tend to get launched in the USA or in Europe. BioBlast has since sold its clinical development to Seelos of New York. Another Israeli biotech is working on a therapy for Duchenne. Please contact Art BioScience – https://www.artbioscience.com/ +972-8-623-7879

      I hope that your nephews have a full recovery.

      Best regards and Shalom

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