Scotland is now screening every newborn for a condition that can kill within two years. Here is what parents across the UK need to know.

A Litchfield woman is running the Boston Marathon to raise awareness for spinal muscular atrophy and honor her daughter, who defied medical expectations by living beyond her second birthday.

Spinal Muscular Atrophy (SMA) causes progressive muscle weakness and, without treatment, can limit life expectancy to just ...

A new qualitative participatory study explored how powered mobility (PM), in the form of modified ride-on cars, impacts children with spinal muscular atrophy type 1 (SMA1) and their families. Here are ...

Hope Scottish pilot will result in heel prick test for rare genetic condition being approved across Britain ...

"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 [email protected]. Topline results from a phase 3 clinical trial showed an ...

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 ...

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 ...

SMA is a genetic condition that impacts motor neurons, leading to muscle weakness that affects essential functions like breathing and movement. Treatments like Spinraza and Zolgensma can help manage ...