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 ...
- Data to highlight latest long-term preclinical efficacy and durability data supporting PBGENE-DMD for the treatment of Duchenne Muscular Dystrophy - - On track to file an IND and/or CTA by the end ...
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 ...
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 ...
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