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FDA approves gene therapy drug for pediatric SMA

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    FDA approves gene therapy drug for pediatric SMA

    This is NOT directly relevant to MND pharmacologically, but it shows that advances are being made elsewhere in gene therapy. But also the cost - $425,000 per year for five years!

    https://www.fda.gov/news-events/pres...y-rare-disease

    https://www.cnbc.com/2019/05/24/fda-...e-therapy.html

    Doug

    #2
    Thanks for posting that, Doug. It's good to see gene therapy making progress. I note that the eye watering price tag is reported to be half of what it would otherwise cost to care for someone with SMA for 10 years.

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