A rapid test using zebrafish was able to show that two children identified by genetic testing as possibly having spinal…
Articles by Marisa Wexler, MS
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…
The European Commission has approved a new high-dose regimen of Spinraza (nusinersen) for people with 5q spinal muscular…
A program that aims to simultaneously strengthen muscles needed for breathing and postural muscles in the trunk may benefit people…
Evrysdi (risdiplam) can stabilize motor function, improve swallowing ability, ease fatigue, and improve life quality in adults with…
A medicine called leuprorelin acetate, approved in the U.S. to treat prostate cancer, can slow the progression of physical disability…
Last month’s approval of Novartis‘ one-time gene therapy Itvisma (onasemnogene abeparvovec-brve) for people with spinal muscular atrophy…
Fatigue in the diaphragm — the large muscle at the base of the chest that pulls air in and out…