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 ...
Since the isolation of the gene that causes Duchenne muscular dystrophy (DMD), scientists have progressed in understanding the mechanisms that lead to muscular diseases that can be evident from the ...
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 ...