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The Food and Drug Administration on Thursday approved a new medicine from Sarepta Therapeutics that treats certain patients with Duchenne muscular dystrophy.

The new drug, called Amondys 45, is Sarepta’s third marketed treatment for the rare, inherited muscle-wasting disease. It will be used to treat the approximately 8% of patients with a confirmed mutation to the Duchenne gene that is amenable to exon 45 skipping.

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  • I’m a person with ALS and just this week the FDA did not approve NurOwn for treatment of ALS. Interesting how the drugs above have been fast tracked through the FDA and yet ALS has seen nothing for new drugs being approved.

    Adam we pALS would love to see you investigate why NurOwn and other such drugs with much better results are not being appoved by the FDA and based on what you’ve written above should all be out there for us pALS to use.

    We need your help!

    Thanks,
    Jim

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