The top-5 most read articles on spinal muscular atrophy (SMA) span novel research in predictive biomarkers, the benefits of newborn screening, and findings that could reshape SMA as a multisystem ...

Panelists discuss how the current spinal muscular atrophy (SMA) treatment landscape includes 3 options: gene therapy (onasemnogene abeparvovec [Zolgensma]) for younger patients and 2 splice modifiers ...

"It's encouraging and it gives me hope that they're finally understanding that we deserve to have treatment just like the rest and that we deserve a chance," she said. "At least a ...

"Decreasing AMMO [active maximal mouth opening] over time is seen most often in patients with SMA type 2 with an estimated reduction of almost 1 mm per year...At this point, we propose that ...

We were unable to process your request. Please try again later. If you continue to have this issue please contact customerservice@slackinc.com. Topline results from a phase 3 clinical trial showed an ...

Please provide your email address to receive an email when new articles are posted on . The study included 58 individuals with SMA types 2 and 3 who were treated with IV apitegromab for 52 weeks. At ...

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 ...

"Our findings suggest that patients with spinal muscular atrophy experience various types of respiratory events, which cannot be differentiated by oximetry. Thus, polysomnography combined with ...

The least-squares mean difference in the Hammersmith Functional Motor Scale-Expanded change from baseline at 12 months was 1.8 points for those 2 to 12 years receiving apitegromab vs placebo.

SMA type 3 is a hereditary disease that affects motor neurons. People may also refer to it as Kugelberg-Welander disease. Symptoms usually begin after 18 months of age. Children with SMA type 3 can ...

Former Little Mix star Jesy Nelson has revealed her twin daughters, Ocean Jade and Story Monroe Nelson-Foster, have been diagnosed with Spinal Muscular Atrophy (SMA) Type 1, a rare genetic condition ...

Patients with Spinal Muscular Atrophy urge the Prime Minister for access to affordable generic Risdiplam to save lives.