FDA approves first drug for spinal muscular atrophy

The U.S. Food and Drug Administration recently approved Spinraza (nusinersen), the first drug approved to treat children and adults with spinal muscular atrophy (SMA). The FDA granted this application fast track designation and priority review. The drug also received orphan drug designation, which provides incentives to assist and encourage the development of drugs for rare diseases.

Thanks to Dr. Nick Willard for alerting me to this development.