rarerelated tag: Phase II

It’s now been more than four years since Spinraza (nusinersen) was first approved by the U.S. Food and Drug Administration, and presymptomatic children with spinal muscular atrophy (SMA) who received the therapy as part of the Phase 2 NURTURE trial are growing up fast and, by all accounts, showing good progress.

Jase Yoder, 5, is one of those children. He was featured as part of “The Gene” documentary on PBS last year. In the segment, Jase, who has SMA type 1, can be seen jumping on a trampoline. Sitting up, walking, and jumping were all milestones Jase would have likely not achieved if it weren’t for Spinraza infusions. In fact, children with SMA type 1 typically don’t make it past the age of 2.