Myotonic Muscular Dystrophy (MMD)

'Gapmer Antisense' Targets MMD1 Defect for Destruction

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).

MMD1: Synthetic 'H' Molecules Lock Up Toxic Repeats

Editor's note 3/15/12: This story was updated to reflect the availability of a podcast with Matthew Disney.

Small, laboratory-designed molecules can make a big difference in cells carrying the genetic defect that causes type 1 myotonic dystrophy (DM1, or MMD1), researchers have found.

MDA Commits $12 Million to Neuromuscular Disease Research

The Muscular Dystrophy Association has awarded 38 new grants totaling more than $12 million to fund research projects focused on its continuing mission to uncover the causes of, and develop therapies for, the more than 40 neuromuscular diseases in its program.

MDA's Board of Directors reviewed and approved the new grants based on recommendations from the Association's Scientific and Medical Advisory Committees, and the grants took effect Feb. 1.

MMD - Laura Ranum, Ph.D.

MDA awarded a research grant totaling $415,092 over a period of three years to Laura Ranum, professor of molecular genetics and microbiology at the University of Florida in Gainesville.

The funds will help support Ranum's research into the role of a phenomenon called Repeat Associated Non-ATG translation (RAN translation) in myotonic dystrophy (MMD, or DM1).

MMD - Mani Mahadevan, M.D.

Mani Mahadevan, a professor at the University of Virginia in Charlottesville was awarded an MDA grant totaling $281,352 over a period of two years. The funds will help support Mahadevan's investigation into potential therapies for type 1 myotonic muscular dystrophy (MMD1, or DM1).

International MMD Consortium Includes Professionals, Families

The 8th International Myotonic Dystrophy Consortium Meeting (IDMC-8), was an exciting mix of the latest scientific developments and clinical research in types 1 and 2 myotonic dystrophy (MMD1 and MMD1, also known as DM1 and DM2).

MMD Research Update Streamed for Families Dec. 3

As part of the upcoming International Myotonic Dystrophy Consortium (IDMC-8) in Clearwater, Fla., a session will be held Saturday, Dec. 3, 2011, to provide a research update for individuals and families affected by myotonic muscular dystrophy (MMD).

Congenital MMD1

Signs and Symptoms

When MMD symptoms manifest at birth, life-threatening complications ensue. However, once this critical period is past, improvement is likely during early childhood. Later, as a child approaches adolescence, it's likely that the symptoms of adult-onset MMD1 will appear.

Adult-Onset MMD1 and MMD2

Signs and Symptoms

The subjects covered in this section are:

MDA Awards $13.7 Million in Research Grants

The Muscular Dystrophy Association has awarded 40 research grants totaling $13.7 million to advance the understanding of disease processes and uncover new strategies for treatments and cures of muscular dystrophy and the more than 40 other diseases in the Association's program.

The new grants were recommended by MDA's Scientific and Medical Advisory Committees and approved by MDA's Board of Directors at its July 2011 meeting.

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