Muscular Dystrophies

Genetics: You, Me and PGD

Update 5/21/12:This study is closed, and results are available at Decision Making About PGD Is Complex, Study Finds. Information about future studies will be posted at PGD: Couples' Decision Making when available.

Preimplantation genetic diagnosis (PGD) is a way for couples to avoid passing along a hereditary disease to their offspring by pre-screening embryos.

Conference Focuses on Becker MD

An MDA-supported conference for families and professionals interested in scientific, medical and social issues related to Becker muscular dystrophy (BMD) will take place Aug. 1, 2009, at Massachusetts General Hospital in Boston.

The one-day conference is free, but seating is limited and attendees must register by July 24.

The program features:

MMD Research: 'Bright' Prospect

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 experiments and human trials in other diseases.

Conference Focuses on Becker MD

An MDA-supported conference for families and professionals interested in scientific, medical and social issues related to Becker muscular dystrophy (BMD) will take place Aug. 1, 2009, at Massachusetts General Hospital in Boston.

The one-day conference is free, but seating is limited and attendees must register by July 24.

The program features:

MMD1 Research: Iplex Shows Limited Benefit

The drug Iplex, developed by the Richmond, Va., biopharmaceutical company Insmed, did not improve muscle function, strength or endurance in a phase 2 trial in type 1 myotonic dystrophy (MMD1, or DM1), the company announced June 25, 2009. (See Insmed Announces Results.)

Iplex Shows Limited Benefit in MMD1

The drug Iplex, developed by the Richmond, Va., biopharmaceutical company Insmed, did not improve muscle function, strength or endurance in a phase 2 trial in type 1 myotonic dystrophy (MMD1, or DM1), the company announced June 25, 2009. (See Insmed Announces Results.)

MMD: Insmed Announces Results

RICHMOND, VA., June 25, 2009 - Insmed Inc. (NASDAQ CM: INSM), a biopharmaceutical company, today announced results from its exploratory U.S. Phase II clinical trial evaluating IPLEX™ (mecasermin rinfabate) in patients with myotonic muscular dystrophy (“MMD”).  The randomized, double-blind, placebo-controlled Phase II trial conducted in 13 centers across the U.S.

MD Research: 3-Protein Repair Cluster

Scientists in the United States and Japan have identified a three-protein cluster that reseals damaged muscle-fiber membranes. The findings, published June 5, 2009, in the Journal of Biological Chemistry, could have implications for development of treatments for muscular dystrophies.

Three-Protein Repair Cluster Identified

Scientists in the United States and Japan have identified a three-protein cluster that reseals damaged muscle-fiber membranes. The findings, published June 5, 2009, in the Journal of Biological Chemistry, could have implications for development of treatments for muscular dystrophies.




MD Research: Muscle-Repair Booster

In experiments in mice, Michael Rudnicki, an MDA grantee at the Sprott Center for Stem Cell Research at Ottawa Hospital Research Institute (OHRI), and colleagues, found the WNT7a protein stimulates muscle repair by causing proliferation (an increase in number) of "satellite stem cells." They say the protein probably operates similarly in humans. The findings were published June 5, 2009, in the journal Cell Stem Cell.

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