Data from the SAPPHIRE study formed the basis of Scholar Rock's U.S. Food and Drug Administration request to approve ...

Unearthing an old stuffed animal led columnist Alyssa Silva to recall the memory of realizing how different her childhood was ...

Columnist Ari Anderson reflects on the memories that made his old house so special and looks forward to making many more in ...

After five years of Evrysdi, people with SMA types 2 and 3 are continuing to show stabilization and improvement in motor ...

Children with SMA given the gene therapy Zolgensma in infancy are maintaining motor milestones after up to 10 years, new ...

Columnist Sherry Toh thanks the dedicated nurses who've attended to her at various hospitals and at home over the years.

This weekend kicked off the MDA annual Clinical & Scientific Conference, bringing together the neuromuscular disease ...

Thanks to the 21st Century Cures Act, columnist Brianna Albers tends to needlessly stress about lab results her doctor hasn't yet reviewed.