ROCKVILLE, Md., Sept. 29, 2025 /PRNewswire/ -- REGENXBIO Inc. (Nasdaq: RGNX) today announced Chief Medical Officer, Steve Pakola, M.D., will present at the ...

Cipaglucosidase alfa/miglustat is an investigational therapy that is not yet approved by any regulatory agency. The posters will be made available on the Amicus website following their respective ...

- Oral presentation of ASPIRO results to include additional efficacy and safety data on all patients in dose Cohort 1 and the first treated patients in dose Cohort 2 - Additional presentations include ...

New Evrysdi five-year data from the SUNFISH study showed continued stabilisation of motor function in a broad population of individuals with Types 2 or 3 spinal muscular atrophy (SMA) Late-breaking ...

Muscle contraction is the tightening, shortening, or lengthening of muscles when you do some activity. It can happen when you hold or pick up something, or when you stretch or exercise with weights.

ROCKVILLE, Md., Sept. 29, 2025 /PRNewswire/ -- REGENXBIO Inc. (RGNX) today announced Chief Medical Officer, Steve Pakola, M.D., will present at the International Congress of the World Muscle Society ...