FDA denies apitegromab approval for SMA, cites manufacturing issues
The U.S. Food and Drug Administration (FDA) has declined to approve apitegromab as a treatment for spinal muscular…
Articles by Marisa Wexler, MS
SMA therapy apitegromab could be available by end of year, if approved
Scholar Rock plans to have the the muscle-strengthening therapy apitegromab available commercially in the U.S. before the…
Treatment with AJ201 shows positive signs in SBMA clinical trial
Treatment with AJ201 was well tolerated and showed signs of improving physical activity in a clinical trial involving people with…
Medicines into amniotic fluid may be way to treat SMA before birth
Injecting medicines into the amniotic fluid — the liquid that surrounds a developing fetus during pregnancy — may be a…
High-carbohydrate diet may help normalize SMA metabolism
A study found that people with spinal muscular atrophy (SMA) have metabolic alterations due to impairments in how their…
Zolgensma gene therapy liver damage usually resolves in months
Liver damage may occur as a side effect of the spinal muscular atrophy (SMA) gene therapy Zolgensma (onasemnogene…
New form of SMA treatment Evrysdi may give patients ‘freedom’
A tablet formulation of Evrysdi (risdiplam) for spinal muscular atrophy (SMA) recently approved in the U.S. is…
MDA 2025: Spinraza treatment stabilizes adult motor function
Treatment with Spinraza (nusinersen) can help stabilize motor function in adults with spinal muscular atrophy (SMA), according to…
MDA 2025: SMA gene therapy ‘transformational,’ per Novartis exec
It’s been almost six years since the one-time gene therapy Zolgensma (onasemnogene abeparvovec-xioi) was first approved in the U.S.
MDA 2025: Apitegromab could fill unmet SMA needs, executive says
The muscle-strengthening therapy apitegromab is being considered for approval by the U.S. Food and Drug Administration (FDA) as an…