Myotonic Muscular Dystrophy (MMD)

Trial of Antisense Drug for Type 1 Myotonic Dystrophy Opens

A phase 1-2 trial of ISIS-DMPKRx, an experimental compound for the treatment of type 1 myotonic muscular dystrophy, is now underway in the U.S. in adults with this disorder who are 20 to 55 years old and meet other study criteria.

MMD/DM - Bernard Jasmin, Ph.D.

Bernard Jasmin, a professor in the Department of Cellular & Molecular Medicine at the University of Ottawa, has been awarded an MDA grant totaling $253,800 over three years to examine the role of a protein called staufen 1 in type 1 myotonic muscular dystrophy (MMD or DM) . In this disorder, staufen 1 interacts with abnormally expanded genetic material in muscle cells.

MMD/DM - Andrew Berglund, Ph.D.

Andrew Berglund, a professor of biochemistry, biophysics and molecular biology at the University of Oregon in Eugene, has been awarded an MDA research grant totaling $253,800 over three years to pursue changing the shape of the abnormal genetic material underlying type 1 and type 2 myotonic muscular dystrophy (MMD or DM).

MMD/DM - Steven Zimmerman, Ph.D.

Steven Zimmerman, a professor of chemistry at the University of Illinois at Urbana-Champaign, has been awarded an MDA research grant totaling $253,800 over three years to develop experimental drugs to treat type 1 myotonic muscular dystrophy (MMD).  Conducting experiments in cells and mice with an MMD-like disorder, Zimmerman and colleagues will test a new compound that keeps the MBNL1 protein away from genetic material called RNA.

MMD1: Antisense Drug Moves to Human Testing


Update (June 11, 2014): This story has been updated with additional information about the phase 1 trial of ISIS-DMPKRx in healthy volunteers and the planned trial in people with type 1 MMD, as well as the availability of a fact sheet on this drug provided by Isis Pharmaceuticals.


Isis Pharmaceuticals Initiates Phase 1 Study of ISIS-DMPK Rx to Treat Myotonic Dystrophy Type 1

Isis Earns $14 Million Milestone Payment from Biogen Idec ISIS-DMPKRx is the Second Generation 2.5 Antisense Drug to Enter the Clinic

New Forms of Genetic Testing Improve Diagnosis, Raise Questions

“Knowing, if not all, is almost all,” said Matthew Harms, a neurologist and neurophysiologist from Washington University in St. Louis, in his presentation on genetic testing for neuromuscular disorders at the 2014 MDA Clinical Conference, held in Chicago March 16-19.

MMD2: Designer Compounds Free a Trapped Protein

Study Seeks Parents, Guardians of Young Children with Neuromuscular Disease

The National Institute of Nursing Research, part of the National Institutes of Health (NIH) in Bethesda, Md., is seeking parents or guardians of children from birth through age 5 with a neuromuscular disease for a study to develop a questionnaire about motor function in young children.

Companies to Develop Antisense Drug for Myotonic Dystrophy

 Biotechnology companies Isis Pharmaceuticals and Biogen Idec have entered into a multi-year collaboration to advance antisense technology for the treatment of neurological diseases — including type 1 myotonic dystrophy (MMD1, or DM1).