Muscular Dystrophy News6d
Corticosteroid at low dose may help with muscle damage, inflammation
A pilot study in 19 LGMD patients and one with Becker MD found low-dose prednisone helped lower markers of muscle damage and ...
Muscular Dystrophy News4d
FDA awards fast track designation to DM1 treatment DYNE-101
The FDA has granted fast track designation to DYNE-101, a therapy for myotonic dystrophy type 1 (DM1) now in testing in a ...
Muscular Dystrophy News12d
DYNE-101 is leading to functional gains for DM1 patients in trial
DYNE-101 eased myotonia and led to other functional improvements for people with myotonic dystrophy type 1 (DM1) in a ...
Muscular Dystrophy News13d
GIVI-MPC named FDA orphan drug for treatment of Becker MD
GIVI-MPC, a stem cell therapy for certain types of muscular dystrophy, has been granted orphan drug status for treating ...
Muscular Dystrophy News4d
What success looks like for me as a DMD parent
As a parent and caregiver to three sons with DMD, columnist Betty Vertin's definition of what success looks like has changed over the years.
Muscular Dystrophy News11d
Stuck in limbo with both accessible vans out of commission
An accident and a malfunction render both of columnist Betty Vertin's accessible vans useless, leaving the family without transportation.
Muscular Dystrophy News14d
GIVI-MPC named FDA orphan drug for treatment of Becker MD
Note: This story was updated Jan. 17, 2025, to clarify the company’s comments on gene therapies, which generally deliver a smaller form of dystrophin. This designation aims to encourage the ...