The first patient has been dosed in a clinical trial testing GB221, a next-generation gene therapy for spinal muscular…
Articles by Marisa Wexler, MS
Spinal muscular atrophy (SMA) is associated with multi-organ changes in a protective protein modification that helps guard cells against…
Treatment with Evrysdi (risdiplam) may modestly improve arm function for adults with spinal muscular atrophy (SMA), a new…
Children with spinal muscular atrophy (SMA) often struggle to get a good night’s sleep — and their caregivers also…
Children with spinal muscular atrophy (SMA) who start on a disease-modifying therapy (DMT) — four have been approved in…
Treatment with a high-dose regimen of Spinraza (nusinersen) was shown to safely improve motor function in children with…
A rapid test using zebrafish was able to show that two children identified by genetic testing as possibly having spinal…
Treatment with gene therapy significantly prolonged survival and improved motor function in a mouse model of spinal muscular atrophy…
For babies diagnosed with spinal muscular atrophy (SMA) before birth, doctors may be able to plan an early delivery…
Spinal muscular atrophy (SMA) affects the nervous system — essentially, the body’s command center, where nerves transmit signals that…