Zolgensma‘s approval as the first gene therapy for treating all forms of spinal muscular atrophy (SMA) in children…
Articles by Emma Yasinski
Preliminary data from two ongoing clinical trials into risdiplam (RG7916) — a potential oral therapy for all types of…
AveXis has filed a first request for approval of its highly-anticipated gene therapy, AVXS-101, to treat infants with spinal muscular atrophy (SMA)…
When Megan Cardenas and her husband brought their 10-day-old son, Derek, to be screened for spinal muscular atrophy (SMA),…