MDA 2025: SMA kids maintaining Zolgensma benefits 10 years later
Children with spinal muscular atrophy (SMA) given the gene therapy Zolgensma (onasemnogene abeparvovec-xioi) in infancy are maintaining motor…
Articles by Marisa Wexler, MS
MDA 2025: SMA gene therapy OAV101 shows acceptable safety
OAV101 IT, a version of the gene therapy Zolgensma (onasemnogene abeparvovec-xioi) that’s administered into the spinal canal, can be…
MDA 2025: Conference begins, marking another year of progress
The Muscular Dystrophy Association‘s (MDA) annual Clinical & Scientific Conference kicked off over the weekend, bringing together patients,…
SMA Europe provides funding for five new research projects in SMA
SMA Europe is funding five new research projects that are intended to advance understanding of spinal muscular atrophy…
Child doing well after mother had SMA treatment while pregnant
The mother of a baby with spinal muscular atrophy (SMA) was given Evrysdi (risdiplam) for the last few…
Scoliosis, kyphosis common with SMA type 1 in DMT era: Study
While effective treatment is lengthening life and improving its quality, children with spinal muscular atrophy (SMA) type 1 often…
FDA approves new tablet version of oral SMA treatment Evrysdi
The U.S. Food and Drug Administration (FDA) has approved a new tablet formulation of Evrysdi (risdiplam), an approved oral treatment…
Cure SMA seeks $10M in DOD funding in advocacy campaign
Cure SMA is leading an advocacy campaign for new research funding from the U.S. Department of Defense (DOD) that…
Scholar Rock asks FDA to approve apitegromab for SMA
Scholar Rock is asking the U.S. Food and Drug Administration (FDA) to approve apitegromab, its experimental add-on therapy…
Model predicts scoliosis risk in patients receiving SMA treatment
An algorithm that takes into account clinical measures of motor symptom severity alongside data on factors like age and height…