Nearly half of the 24 children with myotonic dystrophy type 1 (DM1) in a French study showed signs of…
Articles by Lindsey Shapiro, PhD
Pelvic floor disorders, including urinary and anal incontinence, are common in women with myotonic dystrophy type 1 (DM1) and…
The U.S. Food and Drug Administration (FDA) has placed a hold on clinical testing of ENTR-601-44, Entrada Therapeutics‘ investigational…
Analyzing the type of mutation in the DMD gene combined with assessing motor milestones may help better distinguish Duchenne muscular dystrophy…
The first patient has been dosed in a pilot study evaluating vamorolone as a potential treatment for Becker…
The U.S. Food and Drug Administration (FDA) has given the go-ahead for the first-in-human dosing of Cure Rare Disease’s…
Treatment with dystrophin expressing chimeric (DEC) cells was safe and led to long-term cellular and functional improvements in the heart,…
The first healthy volunteer has been dosed with an investigational exon-skipping therapy, called PGN-EDO51, for Duchenne muscular dystrophy (DMD) in…
The Muscular Dystrophy Association (MDA) has launched its 40th annual MDA Shamrocks fundraising campaign — one of the…