Children with SMA face higher emotional and behavioral challenges
Children with spinal muscular atrophy (SMA) have higher rates of emotional and behavioral challenges than children in the general…
Articles by Marisa Wexler, MS
FDA approves SMA gene therapy Itvisma for patients 2 and older
Before the end of this year, thanks to a new decision by the U.S. Food and Drug Administration (FDA), one-time…
After FDA meeting, Scholar Rock to again seek apitegromab approval
After a “constructive and collaborative in-person” meeting with the U.S. Food and Drug Administration (FDA), Scholar Rock is…
Adding BIO101 to Spinraza boosts muscle function in SMA mice
The experimental therapy BIO101 may help improve muscle strength in people with spinal muscular atrophy (SMA), according to new…
Spinraza may help lung function, delay need for breathing aid
Treatment with Spinraza (nusinersen) may help stabilize lung function in people with spinal muscular atrophy (SMA) types 2…
Study suggests cell communication leads to weak bones in SMA
Mutations that cause spinal muscular atrophy (SMA) may trigger problems with how bone cells communicate, leading to abnormally weakened…
SMA eye movements suggest brain changes, not weakness: Study
People with spinal muscular atrophy (SMA) may show subtle abnormalities in eye movements, but these abnormalities are unlikely to…
Almost all kids given early Evrysdi walking 3 years later: Trial data
Nearly all children with spinal muscular atrophy (SMA) who were started early on Evrysdi (risdiplam) in a clinical…
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…
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…