Spinraza is the first-ever FDA-approved drug for SMA
This life-threatening disease is terrible, but there is hope on the horizon. The Federal Drug Administration has approved Spinraza, which is the first-ever FDA-approved therapy for SMA, in December 2016. The drug has received the orphan drug designation from the United States and the European Union, meaning it is approved for rare diseases like SMA. While the drug is still new and there is more research to be done, this is definitely helpful in finding an eventual cure for this tragic disease.