Up to a year of treatment with DYNE-251, Dyne Therapeutics’ investigational exon 51-skipping therapy, led to improvements in motor…
Articles by Lindsey Shapiro, PhD
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…
Dosing has been paused in a Phase 3 trial testing fordadistrogene movaparvovec in children with Duchenne muscular dystrophy…
Agamree (vamorolone) is now available for Duchenne muscular dystrophy (DMD) patients, ages 2 and older, in the U.S.,…