FDA approves gene therapy drug for pediatric SMA

[Doug Carpenter]

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

[johnburton]

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.