Muscular Dystrophies

Abnormal Activation

An MDA-supported team of scientists in the United States and the Netherlands has uncovered new leads about the origins of facioscapulohumeral muscular dystrophy (FSHD), a disease whose biochemical underpinnings have proved elusive to scientists despite years of investigation.

Lack of understanding of the mechanisms involved in FSHD has impeded treatment development, a phase of research that generally moves forward after disease mechanisms have been described.

Congenital MD Meeting

A conference for families affected by congenital muscular dystrophy (CMD) is taking place Saturday and Sunday, Aug. 15-16, 2009, at Children's Hospital of Philadelphia, under the sponsorship of Cure CMD.

The conference begins at 8:30 a.m. Eastern time and will be available through the Internet.

Iplex on Hold

The Richmond, Va., biopharmaceutical company Insmed announced July 27, 2009, that it will not supply its experimental drug Iplex to any new patients with amyotrophic lateral sclerosis (ALS) for the foreseeable future, and that it intends to analyze the available data on Iplex for ALS and type 1 myotonic dystrophy (MMD1, or DM1) before deciding whether to proceed with development of the drug for either dise

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.

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