Myotonic Muscular Dystrophy (MMD)

MMD Research: Disrupted Disease Process

 

A compound that has the potential to be refined and modified into a treatment for type 1 myotonic dystrophy (MMD1, or DM1) has been identified by researchers at the University of Oregon in Eugene, and the University of Rochester (N.Y.) School of Medicine and Dentistry.

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

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.

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.

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.

Silencing Toxic Genes

A new gene therapy approach to "silencing" disease-causing genetic information has been developed by researchers at Rutgers University in Piscataway, N.J., and Integrated DNA Technologies in Coralville, Ia.

MMD Research: Drug Development?

The identification of small molecules that can block the genetic defect that causes type 1 myotonic dystrophy (MMD1, or DM1) may be the first step toward developing a new drug treatment for the disease, say researchers at the University of Rochester (N.Y.) Medical Center (URMC).

The abnormality that underlies MMD1 is a stretch of genetic material derived from DNA on chromosome 19 that contains more than the usual number of a repeating chemical sequence known as a CUG (cytosine, uracil, guanine) triplet repeat.

Pages