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The Food and Drug Administration reversed its decision on a treatment for Duchenne muscular dystrophy from Sarepta Therapeutics, approving a previously rejected drug without explaining what the problem was in the first place.

The therapy, called Vyondys 53, is approved to treat the roughly 8% of Duchenne patients whose disease results from a specific DNA error. In a clinical trial, Sarepta’s drug produced a small increase in an important muscle protein called dystrophin that is normally missing in children with Duchenne. The company has yet to demonstrate that Vyondys 53 can improve muscle function or slow the progression of the disease.

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  • The cost of this medication will cause financial hardship for patients families along with the huge cost of caring for these children. What will the company and the government do so this drug is available!

  • Meu nome é dayana tenho 32 anos tenho distrofia muscular de cintura e gostaria de saber se esse tratamento pode ser feito em paciente com distrofia muscular de cintura quero ter força de volta peço que quero ser cobaia em suas pesquisas eu acredito muito nas pesquisas de vocês e não aguento mais não ter força e não poder fazer nada me ajuda por favor quero andar de novo e meu sonho é dançar aguardo sua respostas.
    Não quero perder minha vida por causa dessa doença.

  • in June 2019 the FDA rejected Edsivo for the treatment of Vascular Ehler Danlos Syndrome (vEDS). Edsivo (Celiprolol) is currently the standard of care in Europe for vEDS, and while the clinical data was imperfect (ironically, the trial was halted due to efficacy),a subsequent database did incorporate a large proportion of all vEDS patients in France showing actual clinical benefit for this population.
    It should be noted that vEDS is often lethal and quite debilitating, causing sudden ruptures in blood vessels.
    So tell me why does the FDA approve two therapies based on small biomarker trials and reject as comprehensive a data set as can be obtained in an orphan indication? If there was a clear policy, fine. But it’s clear there isn’t. At least when it comes to clear standards in orphan approvals, the FDA has lost its way.
    Also, it would be great if STAT looked at this.

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