Note: This story was updated March 31, 2023, to clarify that the presentation was given at the Muscular Dystrophy Association’s…
Articles by Marisa Wexler, MS
Four boys with Duchenne muscular dystrophy (DMD) who were treated with the gene therapy candidate SRP-9001 (delandistrogene moxeparvovec) in…
People with Duchenne muscular dystrophy (DMD) who are treated with Exondys 51 (eteplirsen) live significantly longer than would…
Researchers are planning to enroll a new group of patients in a natural history study to collect data about how…
A new gene editing technique was able to fix the mutation that most commonly causes type 2A limb-girdle muscular…
Protecting structures on the end of chromosomes, called telomeres, by increasing levels of the protein TRF2, was found to improve…
A new dominant-negative tumor necrosis factor (DN-TNF) treatment reduced muscle damage and promoted muscle regeneration in a mouse model of…
After 18 months, treatment with the experimental cell therapy CAP-1002 continues to show beneficial effects on upper limb function…
Wave Life Sciences‘ experimental exon-skipping therapy WVE-N531 appears to be working as expected, according to early data from…
The U.S. Food and Drug Administration (FDA) has given priority review Sarepta Therapeutics‘ application seeking accelerated approval of SRP-9001 (delandistrogene…