Charles Thornton, a professor of neurology at the University of Rochester (N.Y.), has received MDA support for research in myotonic muscular dystrophy (MMD, also known as DM) and other neuromuscular diseases. He's currently developing antisense oligonucleotides and small molecules for MMD. Thornton also co-directs the MDA clinic and directs the MDA/ALS Center at the University of Rochester...
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).
A small-molecule compound counteracts some of the effects of abnormal genetic instructions in MMD1
A compound that has the potential to be refined and modified into a treatment for type 1 myotonic dystrophy (MMD1, or DM1) has been identified by researchers at the University of Oregon in Eugene, and the University of Rochester (N.Y.) School of Medicine and Dentistry.
Compound frees a crucial protein from a cellular trap in mice with MMD1; treatment prospects 'bright'
Researchers at the University of Rochester (N.Y.) Wellstone Muscular Dystrophy Cooperative Research Center have identified a compound that has the potential to be developed into a treatment for type 1 myotonic dystrophy (MMD1, or DM1).
The compound, dubbed CAG25, is an "antisense oligonucleotide," a type of construct that's been used to block disease-causing genetic instructions in laboratory...
Story includes research items about: Charcot-Marie-Tooth disease, congenital muscular dystrophy, Duchenne muscular dystrophy, Emery-Dreifuss muscular dystrophy (EDMD), type 1 myotonic muscular dystrophy, and spinal muscular atrophy - including, type 3 SMA and spinal-bulbar muscular atrophy (SBMA, Kennedy disease).