Apitegromab improved motor function in SMA patients in a Phase 3 trial analysis, with stronger benefits seen in those treated earlier.

Biogen has launched a trio of Phase 3 trials to test salanersen, a new SMA treatment that aims to be a more convenient ...

Spinraza treatment led to sustained improvements in motor function in adults with SMA, according to a South Korean study.

For columnist Sherry Toh, the new TV series "The Beauty" dwells too much on the grief of living with a rare disease but ignores the strength.

Scholar Rock plans to resubmit its application seeking approval for apitegromab, an investigational muscle-strengthening therapy for SMA.