The complex and multifaceted disease known as myotonic muscular dystrophy (MMD) — also known as dystrophia myotonia (DM) — was the subject of an In Focus report in the April-June 2012 Quest.
Here, we delve into experimental strategies that may markedly improve the outlook for people with this disorder.
Thomas Cooper, a professor in the Department of Pathology and Immunology at Baylor College of Medicine, is a longtime MDA research grantee who has current MDA support to develop oligonucleotide-based therapies for myotonic muscular dystrophy (MMD, also known as DM).
Margaret Wahl, MDA's medical and science editor, talked with Cooper about his research.
Researchers have used a 'gapmer antisense' strategy to destroy the genetic defect that causes type 1 myotonic dystrophy in cultured cells and in MMD1 mice
Researchers at Baylor College of Medicine in Houston and Isis Pharmaceuticals in Carlsbad, Calif., have announced encouraging results for their antisense-based strategy in development for the treatment of type 1 myotonic muscular dystrophy (DM1, or MMD1).