Forbes contributors publish independent expert analyses and insights. Spinal muscular atrophy affects the nerves that control muscle movement, leading to progressive weakening. As a result, infants ...

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 ...

A positive newborn screening for spinal muscular atrophy (SMA) is currently considered a medical emergency. Without early treatment, severe disability or death in infancy are likely. However, research ...

Spinal bulbar muscular atrophy (SBMA) causes a loss of motor neurons in the spinal cord and brainstem. It mainly affects facial and swallowing muscles and the muscles in the arms and legs. Previously, ...

Spinal muscular atrophy (SMA) is a rare genetic condition that causes progressive muscle weakness, which, when untreated, prevents infants with the most severe form from gaining motor ...

Please provide your email address to receive an email when new articles are posted on . At 5 years, 91% of pediatric patients treated with Evrysdi were alive, 81% without permanent ventilation. Data ...

University of Pittsburgh School of Medicine researchers carrying out a small pilot clinical trial demonstrating that a drug-free, minimally invasive intervention targets the root cause of progressive ...

Adult-onset spinal muscular atrophy (SMA) is a rare neuromuscular disorder that primarily affects the motor neurons in the spinal cord, leading to muscle weakness and atrophy. SMA is part of a group ...

A new report provided updated figures estimated the birth prevalence of spinal muscular atrophy (SMA) in the US. Previous investigations revealed that SMA is diagnosed per every 1 in 10,000 births ...

Electrical stimulation of the spinal cord strengthened the muscles of three people with spinal muscular atrophy, a rare motor neuron disease. An experimental treatment that stimulates the spinal cord ...