PBGENE-DMD has received Investigational New Drug clearance from the U.S. Food and Drug Administration (FDA) enabling initiation of clinical trial site activation. PBGENE-DMD was previously granted FDA ...
Striated muscle hamartomas are rare, benign malformations of disorganised but mature striated muscle fibres. This case report presents a congenital striated muscle hamartoma (CSMH) in a primary school ...
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 ...
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 ...
- 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 ...
ClC-1 is a chloride ion channel specifically expressed in skeletal muscle cells that normally dampens muscle fiber excitability and is involved in regulating muscle fiber excitability during exercise.
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