Problems with chemical messengers like dopamine were tied to posture issues in SMA in a mouse study, but levodopa therapy may ease symptoms.

It was a busy week for the biotech sector, with regulatory and pipeline updates. Sage Therapeutics SAGE was in focus after it ...

Scholar Rock submitted its BLA of apitegromab for the treatment of patients with Spinal Muscular Atrophy with a request for Priority Review to be given. Explore more details here.