FDA-approved treatments for spinal muscular atrophy differ in their mechanisms of action, safety profiles, and administration challenges, with key selection factors like patient age and gene copy ...

Evrysdi can stabilize motor function, improve swallowing ability, ease fatigue, and improve life quality for adults with SMA, ...

A Paediatric neurologist shares insight into the condition after former Little Mix singer Jesy Nelson revealed her twins have ...

A retrospective, exploratory analysis identifies biomarkers predictive of spinal muscular atrophy (SMA) response to nusinersen and suggests the efficacy of using machine learning algorithms to ...

The lab of Yongchao C. Ma, PhD, at Stanley Manne Children’s Research Institute at Ann & Robert H. Lurie Children’s Hospital of Chicago uncovered a novel mechanism that leads to motor neuron ...

After up to 4.8 years of continuous treatment with SPINRAZA, 100 percent of children treated pre-symptomatically were alive, and none require permanent ventilation Patients continued to maintain and ...

First and only muscle-targeted SMA therapy to demonstrate statistically significant, clinically meaningful improvements on the gold-standard HFMSE scale (p=0.019) versus placebo, with consistent ...

In a hopeful step for medicine, a 2-and-a-half-year-old child born with spinal muscular atrophy (SMA) has shown no symptoms ...

Co-first and co-corresponding author Richard Finkel, MD, St. Jude Center for Experimental Neurotherapeutics director and Department of Pediatric Medicine member. “The impact of giving risdiplam soon ...

For those of you that don't know what SMA 1 is, it is the most severe muscular disease that a baby can get," Jesy Nelson said ...