STAT1d
Regenxbio says gene therapy strengthened boys with Duchenne muscular dystrophy
Based on a small trial with positive muscle performance data, Regenxbio is expected to pursue approval of its gene therapy ...
Cochrane1d
Medicines that increase alertness (psychostimulants) for excessive daytime sleepiness (hypersomnia) in myotonic dystrophy
Myotonic dystrophy is an inherited muscular dystrophy causing muscle weakness and wasting. Excessive daytime sleepiness (hypersomnia) is a frequent complaint of people with myotonic dystrophy and is ...
Benefit held for boy with aggressive form of Muscular Dystrophy
DICKSON CITY, Pa. — The Eagle Hose Company in Dickson City on Saturday, November 16 hosted a benefit for the Hetzel family as ...
GEN4d
Gene Therapy for Muscular Dystrophy Could Use StitchR to Assemble XL Gene from S Segments
StitchR makes it possible to deliver larger payloads with current vectors, expanding the list of diseases targetable by gene therapies.
Discovery enables effective use of gene therapy for muscular dystrophies and other large-gene diseases
Gene therapy can effectively treat various diseases, but for some debilitating conditions like muscular dystrophies there is ...