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.