Risdiplam, an oral pre–messenger RNA splicing modifier, is an efficacious treatment for persons with symptomatic spinal muscular atrophy (SMA). The safety and efficacy of risdiplam in presymptomatic ...

Distal spinal muscular atrophy (DSMA) is a rare genetic disease that causes a loss of muscle movement. It affects muscles further away from the center of the body, such as the hands, feet, and legs.

In a retrospective analysis, a U.S.-based team of experts recently reported that health-related quality of life (HRQoL) after surgery for early-onset scoliosis (EOS) varies significantly depending on ...

Regina Trollmann, MD, of the Division of Pediatric Neurology, Department of Pediatrics, Friedrich-Alexander-University of Erlangen-Nürnberg, Erlangen, Germany, and colleagues, did a retrospective ...

Spinal muscular atrophy (SMA) is a genetic neuromuscular disease affecting specialized nerve cells that control voluntary muscle movement, according to the Muscular Dystrophy Association (MDA). It can ...

Oral risdiplam (Evrysdi, Genentech) started in the first 6 weeks of life let most infants with presymptomatic spinal muscular atrophy (SMA) reach motor milestones typical of healthy babies, results of ...

Spinal muscular atrophy with respiratory distress (SMARD) is a rare genetic condition that typically affects infants and children. It causes muscle weakness and breathing problems. Spinal muscular ...

British singer Jesy Nelson recently took to Instagram to share a devastating update about her 8-month-old twin daughters.

Taiwanese company AnnJi Pharmaceutical has reported encouraging outcomes from its randomised, placebo-controlled Phase I/IIa trial of AJ201, an investigational compound for treating the adult ...

The U.S. Food and Drug Administration has approved Itvisma (onasemnogene abeparvovec-brve) for the treatment of spinal muscular atrophy (SMA) in patients 2 years and older with confirmed mutation in ...