The SBMA therapy AJ201 showed good safety, hints of efficacy, and improved physical activity for this SMA type in a trial, ...

Injecting medicines into amniotic fluid may be a viable strategy for treating SMA before birth, according to a new study in ...

Making a situation accessible is more about mindset than things, says columnist. That's why attitude is important.

SMA gene therapy GC101 was found to rescue disease manifestations in mice and to be safe in non-human primates.

Blood creatinine-to-cystatin-c ratio, an indicator of muscle mass and strength, may be a treatment response biomarker of SMA, ...

A wearable robotic device called HAL improved walking ability for SMA patients who also received Spinraza (nusinersen), a ...

Spoiler alert: This column discusses the plot of the 2024 film “The Substance.” Hollywood was abuzz about Coralie Fargeat’s 2024 film “The Substance” when it arrived on the scene. It was a ...