Precision BioSciences, Inc. (Nasdaq: DTIL), a clinical stage gene editing company utilizing its novel proprietary ARCUS® ...
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 ...
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.
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 ...
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 ...
BOSTON--(BUSINESS WIRE)-- PepGen Inc. (PEPG), a clinical-stage biotechnology company advancing the next generation of oligonucleotide therapies with the goal of transforming the treatment of severe ...
Some results have been hidden because they may be inaccessible to you
Show inaccessible results