Small molecules that induce or reverse effects of the type 1 myotonic dystrophy mutation in cells in the lab are likely to speed research
Matthew Disney is a current and former MDA grantee at the Scripps Research Institute in Jupiter, Fla. Disney's current MDA grant is focused on targeting toxic RNA in type 2 myotonic dystrophy (MMD2, or DM2).
Located around the United States, MDA’s new network provides a foundation for the conduct of clinical trials in MMD1 and MMD2
MDA has launched a new, five-center clinical research network focused on type 1 and type 2 myotonic muscular dystrophy (MMD1 and MMD2, also known as DM1 and DM2), with the principal goal of preparing for testing of new MMD treatments as they become available.
Two companies will jointly develop a drug for type 1 myotonic dystrophy; UK survey responders say modafinil's benefits for MMD1-related sleepiness outweigh side effects
Type 1 myotonic dystrophy (MMD1, or DM1) and type 2 myotonic dystrophy (MMD2, or DM2) are complex, multisystem disorders caused by similar genetic flaws on chromosome 19 (MMD1) and chromosome 3 (MMD2). Treatments that target the underlying molecular causes of MMD1 and MMD2 are in development.
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.
Matthew Disney is an associate professor in the Department of Chemistry at the Scripps Research Institute in Jupiter, Fla. He has MDA support to design small molecules that target the toxic RNA that underlies myotonic muscular dystrophy (MMD, also known as DM).
MDA Medical and Science Editor Margaret Wahl talked with Matthew Disney about his work.
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...