Treatment with exon 53-skipping therapy WVE-N531 led to significant increases in muscle dystrophin levels along with signs of improved muscle…
Articles by Lindsey Shapiro, PhD
Capricor Therapeutics will soon begin filing an application seeking U.S. approval of its cell therapy deramiocel for cardiomyopathy, a…
Up to a year of treatment with DYNE-251, Dyne Therapeutics’ investigational exon 51-skipping therapy, led to improvements in motor…
A little over a year ago, neurologist Sarah Wright administered Elevidys (delandistrogene moxeparvovec-rokl) to then 5-year-old Hiram Secrist, making…
The experimental gene therapy RGX-202 has been found to be well tolerated at a high dose, with biomarker data…
A low dose of PGN-ED051, PepGen’s investigational exon 51-skipping therapy, safely increased dystrophin protein levels in people with…
Pfizer has discontinued development of fordadistrogene movaparvovec, its investigational gene therapy for Duchenne muscular dystrophy (DMD), after…
A pair of identical twins with myotonic dystrophy type 1 (DM1) were found to have different deficits in social cognition…
DYNE-101 and DYNE-251, Dyne Therapeutics’ investigational therapies for forms of muscular dystrophy, continue to work as intended and…
Findings from a Phase 2 trial testing losmapimod in adults with facioscapulohumeral muscular dystrophy (FSHD) have offered new…