FDA approves first drug for spinal muscular atrophy - Spinraza

December 23, 2016 The U.S. Food and Drug Administration approved Spinraza (nusinersen), the first drug approved to treat children and adults with spinal muscular atrophy (SMA), a rare and often fatal genetic disease affecting muscle strength and movement. Spinraza is an injection administered into the fluid surrounding the spinal cord.

This video explains more about Spinraza and how it works:

We are excited for the opportunity to try this new treatment but are patiently waiting as the hospitals around us get set up.