we propose that there are at least 4 clinically and genetically distinct forms of X-linked muscular dystrophy. IV-6 (A.C.), born in 1927, was not examined. He died of unrelated congenital heart ...
In addition, the infant exhibited pronounced muscular hypotonia (“floppy ... This strengthened the suspicion of congenital myotonic dystrophy type 1 (DM1) in the newborn. Autosomal dominant ...
At the Breakthroughs in Muscular Dystrophy special meeting held in Chicago Nov. 19-20, 2024, and organized by the American Society of Gene & Cell Therapy (ASGCT), multiple interventions at the RNA ...