Last month’s approval of Novartis‘ one-time gene therapy Itvisma (onasemnogene abeparvovec-brve) for people with spinal muscular atrophy…
Articles by Marisa Wexler, MS
Fatigue in the diaphragm — the large muscle at the base of the chest that pulls air in and out…
Last week’s U.S. Food and Drug Administration (FDA) approval of Novartis‘ gene therapy Itvisma (onasemnogene abeparvovec-brve) means one-time…
Children with spinal muscular atrophy (SMA) have higher rates of emotional and behavioral challenges than children in the general…
Before the end of this year, thanks to a new decision by the U.S. Food and Drug Administration (FDA), one-time…
After a “constructive and collaborative in-person” meeting with the U.S. Food and Drug Administration (FDA), Scholar Rock is…
The experimental therapy BIO101 may help improve muscle strength in people with spinal muscular atrophy (SMA), according to new…
Treatment with Spinraza (nusinersen) may help stabilize lung function in people with spinal muscular atrophy (SMA) types 2…
Mutations that cause spinal muscular atrophy (SMA) may trigger problems with how bone cells communicate, leading to abnormally weakened…
People with spinal muscular atrophy (SMA) may show subtle abnormalities in eye movements, but these abnormalities are unlikely to…