Treatment with Spinraza (nusinersen) can help stabilize motor function in adults with spinal muscular atrophy (SMA), according to…
Articles by Marisa Wexler, MS
It’s been almost six years since the one-time gene therapy Zolgensma (onasemnogene abeparvovec-xioi) was first approved in the U.S.
The muscle-strengthening therapy apitegromab is being considered for approval by the U.S. Food and Drug Administration (FDA) as an…
Activating a protein called potassium channel Kv2.1 may help improve motor function in spinal muscular atrophy (SMA), according to…
A higher dose of Spinraza (nusinersen) may be more effective than the currently approved dosing schedule in people with…
Apitegromab, a muscle-strengthening therapy up for approval in the U.S., led to gains in motor function in a clinical…
After five years of treatment with Evrysdi (risdiplam), people with spinal muscular atrophy (SMA) types 2 and 3…
Children with spinal muscular atrophy (SMA) given the gene therapy Zolgensma (onasemnogene abeparvovec-xioi) in infancy are maintaining motor…
OAV101 IT, a version of the gene therapy Zolgensma (onasemnogene abeparvovec-xioi) that’s administered into the spinal canal, can be…
The Muscular Dystrophy Association‘s (MDA) annual Clinical & Scientific Conference kicked off over the weekend, bringing together patients,…