Myotonic Dystrophy News and Research

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MDA awards over $21M in grants for advancing critical neuromuscular research

Forty-two medical researchers and their labs have been awarded more than $21 million in grants by the Muscular Dystrophy Association to advance critical neuromuscular research in 2010. Many of the grants are multi-year awards to be dispersed over the next three years.

15 Apr 2010

Loss of key proteins may account for molecular abnormalities associated with myotonic dystrophy

Research on the genetic defect that causes myotonic muscular dystrophy has revealed that the mutation disrupts an array of metabolic pathways in muscle cells through its effects on two key proteins.

24 Jan 2010

Researchers find compound that reverses type 1 myotonic dystrophy

A compound already used to treat pneumonia could become a new therapy for an inherited muscular wasting disease, according to researchers at the University of Oregon and the University of Rochester School of Medicine and Dentistry in New York.

7 Nov 2009

Small molecule blocks aberrant pathway associated with myotonic dystrophy

Researchers at the University of Illinois have designed a small molecule that blocks an aberrant pathway associated with myotonic dystrophy type 1, the most common form of muscular dystrophy.

8 Sep 2009

New approach to remove toxic agent that causes myotonic dystrophy

Cell biologists from the Radboud University Nijmegen Medical Centre (Nijmegen, the Netherlands) describe a new approach to remove the toxic agent that causes the neuromuscular disease myotonic dystrophy. Their findings are published in the scientific journal The Proceedings of the National Academy of Sciences.

11 Aug 2009

Chemists rationally design inhibitors against an RNA molecule that causes myotonic muscular dystrophy

Chemists at the University at Buffalo have used rational drug design to synthesize small, cell-permeable molecules that are effective in vitro against two common types of myotonic muscular dystrophy, a result that has implications for potentially curing muscular dystrophy, as well as other diseases.

9 Aug 2009

Genetic source of muscular dystrophy blocked

Researchers at the University of Rochester Medical Center have found a way to block the genetic flaw at the heart of a common form of muscular dystrophy. The results of the study, which were published today in the journal Science, could pave the way for new therapies that essentially reverse the symptoms of the disease.

17 Jul 2009

Small molecules might block mutant protein production in Huntington's disease

Molecules that selectively interfere with protein production can stop human cells from making the abnormal molecules that cause Huntington's disease, researchers at UT Southwestern Medical Center have found.

5 May 2009

Small molecules might block mutant protein production in Huntington's disease

3 May 2009

Alternative splicing proteins prompt heart development

Just as the emotions it represents are dynamic, the heart's development requires dynamic shifts in proteins that prompt alternative spicing, a mechanism that allows a given gene to program the cell to make several proteins, said a group of researchers at Baylor College of Medicine in a report that appears online in the journal Proceedings of the National Academy of Sciences.

8 Dec 2008

Scientists discover new compounds to target muscular dystrophy

Using a drug-discovery technique in which molecules compete against each other for access to the target - the strand of toxic RNA that causes the most common form of muscular dystrophy in adults - a team at the University of Rochester Medical Center has identified several compounds that, in the laboratory, block the unwanted coupling of two molecules that is at the root of the disease.

19 Nov 2008

Risk factors for sudden death for adult muscular dystrophy identified

The largest assessment of people with adult muscular dystrophy has identified risk factors that can lead to sudden death for individuals with the most common form of this disease.

19 Jun 2008

RNA toxicity contributes to neurodegenerative disease

Expanding on prior research performed at the University of Pennsylvania, Penn biologists have determined that faulty RNA, the blueprint that creates mutated, toxic proteins, contributes to a family of neurodegenerative disorders in humans.

27 May 2008

RNA toxicity contributes to neurodegenerative disease

21 May 2008

Practice parameters discuss treatment for narcolepsy, other hypersomnias of central origin

Practice parameters published in the December 1 issue of the journal SLEEP serve as both an update of previous practice parameters for the therapy of narcolepsy and as the first practice parameters to address treatment of other hypersomnias of central origin, including idiopathic hypersomnia, recurrent hypersomnia and hypersomnia due to medical condition.

3 Dec 2007

Researchers identify compound that eliminates myotonia - a symptom of muscular dystrophy

Researchers at the University of Rochester Medical Center (URMC) have identified a compound that eliminates myotonia - a symptom of muscular dystrophy - in mice. The research was published online in the Journal of Clinical Investigation.

16 Nov 2007

ClC-1 helps mice get a move on

In addition to the progressive muscle weakness and degeneration common to all forms of muscular dystrophy, patients with the most common form of muscular dystrophy affecting adults, myotonic dystrophy type 1 (DM1), also experience an inability to voluntarily relax the muscles (a problem known as myotonia).

16 Nov 2007