MDA 2025: Potassium channel could be SMA treatment target
Activating a protein called potassium channel Kv2.1 may help improve motor function in spinal muscular atrophy (SMA), according to…
Articles by Marisa Wexler, MS
MDA 2025: High-dose Spinraza safe, effective in DEVOTE study
A higher dose of Spinraza (nusinersen) may be more effective than the currently approved dosing schedule in people with…
MDA 2025: Apitegromab safely improves SMA motor function
Apitegromab, a muscle-strengthening therapy up for approval in the U.S., led to gains in motor function in a clinical…
MDA 2025: Evrysdi consistent after 5-year SUNFISH follow-up
After five years of treatment with Evrysdi (risdiplam), people with spinal muscular atrophy (SMA) types 2 and 3…
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…
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…