Children with spinal muscular atrophy (SMA) who have spine deformities or weak trunk muscles are more likely than others…
Articles by Marisa Wexler, MS
Novartis has completed negotiations with the pan-Canadian Pharmaceutical Alliance (pCPA) regarding pricing and availability of Zolgensma (onasemnogene abeparvovec), its…
A pilot newborn screening (NBS) program for spinal muscular atrophy (SMA) that started in Liège province has transitioned into an…
Scholar Rock plans to soon initiate a Phase 3 clinical trial of apitegromab, its investigational muscle-directed treatment for …
After being treated with Evrysdi (risdiplam) for at least a year, pre-symptomatic infants with spinal muscular atrophy (SMA)…
A naturally occurring form of the androgen receptor protein — which scientists have named AR45 because of its…
The first-ever case of someone in Romania with spinal muscular atrophy (SMA) caused by a mutation in the ASAH1…
The U.S. Food and Drug Administration (FDA) has lifted its partial hold on clinical trials of OAV-101 (AVXS-101), an…
Biochemical modifications made to antisense oligonucleotides — molecules that are the basis of an existing therapy for spinal muscular…
Eurordis opened a campaign, called Rare 2030 Action, that is seeking to establish a European action plan for…