Researchers are planning to enroll a new group of patients in a natural history study to collect data about how…
Articles by Marisa Wexler, MS
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…
PGN-EDO53, an experimental therapy designed to treat Duchenne muscular dystrophy (DMD) in patients amenable to exon 53 skipping, showed…
Sarepta Therapeutics plans to begin a clinical trial to test the antibody-cleaving therapy imlifidase as a pre-treatment for SRP-9001…
Santhera and ReveraGen have completed their rolling application asking the U.S. Food and Drug Administration (FDA) to approve the dissociative…