Chugai Pharmaceutical, part of the Roche Group, is asking that risdiplam be approved as a daily oral treatment for …
Articles by Marisa Wexler, MS
Adults with spinal muscular atrophy (SMA) who began being seen at specialty centers after the approval of Spinraza…
England’s National Institute for Health and Care Excellence (NICE) is expanding its appraisal of Zolgensma, a gene therapy for…
A clinical trial testing oral amifampridine phosphate in people with spinal muscular atrophy (SMA) type 3 who are able to walk is…
Identifying those spinal muscular atrophy (SMA) patients who will benefit most from treatment with Zolgensma requires more than assessing traditional…
Perisynaptic Schwann cells — a specialized type of cell that helps facilitate communication between neurons and muscle cells…
Perceptions about illness often don’t line up between children with spinal muscular atrophy (SMA) and their parents, with mothers…
A readiness program to help clinical trial sites prepare to run spinal muscular atrophy (SMA) studies has been established by…
Siblings of people with spinal muscular atrophy (SMA) who do not have the disease themselves tend to learn about…
A better understanding of the needs of adults with spinal muscular atrophy (SMA), best ensuring for their mental and social…