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…
Articles by Marisa Wexler, MS
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…
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…