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#elevidys — Public Fediverse posts

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  1. Wesley Bond (OLD ACCOUNT) @[email protected] ·

    Very disappointing news on the Duchenne muscular dystrophy front. Elevidys, the only available gene therapy for DMD has shown no significant improvement in motor function in kids receiving the treatment after 1 year. However, certain secondary outcomes showed some benefit.

    It remains to be seen if FDA will allow it to stay on the market in the US, given that there are no other available treatments for DMD.

    ajmc.com/view/sarepta-joins-pf

    #Duchenne #musculardystrophy #raredisease #genetherapy #elevidys #biotech #pharma

    #duchenne #musculardystrophy #raredisease #genetherapy #elevidys #biotech
  2. IT News @[email protected] ·

    Top FDA official overrules staff to approve gene therapy that failed trial - Enlarge / Dr. Peter Marks, Director of the Center for Biologics Evaluat... - arstechnica.com/?p=2032919 #duchennemusculardystrophy #clinicaltrials #clinicaldata #fdaapproval #genetherapy #petermarks #elevidys #science #sarepta #fda

    #fda #sarepta #science #elevidys #petermarks #genetherapy
  3. Brittany Trang @[email protected] ·

    Duchenne muscular dystrophy moms take comfort in the idea that #Elevidys means future DMD parents won't have the stark prognosis they did:

    "There’s no treatment, take them home, love them, come back when they need wheelchairs."

    today's cry, via @STAT's Jason Mast:
    statnews.com/2023/06/30/duchen

    #elevidys