A report on drug development in neuromuscular diseases as of January 2009
posted on January 1, 2009 - 2:37pm
In the era of molecular biology, the drug development process has moved from a “let’s try it and see what happens” approach to a scientifically based process of discovery and application.
For many of the diseases in MDA’s program, drug discovery begins with gene discovery — identifying a gene that, when flawed, causes a disease.
Can toxic genes be blocked to treat disease?
posted on January 1, 2007 - 3:02pm
Since the 1990s, gene therapy - the insertion of functional genes to compensate for nonfunctional ones - has been the goal of researchers working in several muscular dystrophies, spinal muscular atrophy, Friedreich's ataxia, metabolic muscle diseases and myotubular myopathy.
posted on March 1, 2007 - 9:59am
In 1990, Sara Winokur was a doctoral student in the laboratory of John Wasmuth, a professor of biological chemistry and a prominent researcher in genetics at the University of California at Irvine.
It was an exciting time in genetics. The genes that, when mutated (flawed), cause diseases, were rapidly being identified. Among the first, in 1986, had been one for Duchenne muscular dystrophy.
Duchenne muscular dystrophy carriers carry on, despite uncertainty
posted on November 1, 2007 - 4:20pm
When Rena Szymanski turned 40, she expected to slow down a bit. She never had been athletic, and it sometimes seemed to her that climbing stairs was harder for her than it was for other people, but she thought her strength was “normal” in general.
Soon after her birthday, though, she noticed increasing weakness. “I didn’t know what was wrong with me,” says Szymanski, who until recently was...
An antibody from llamas, tested in fruit flies, holds promise as a treatment for OPMD
posted on March 26, 2009 - 9:00pm
Scientists in France and the Netherlands recently announced they've identified a promising new strategy that could potentially become a therapy for oculopharyngeal muscular dystrophy (OPMD), a form of MD that primarily weakens the eyelid and throat muscles and also can affect muscles in the limbs.
The strategy involves using an antibody (immune-system protein) derived from llamas. The antibody...
MMD, FSHD, OPMD, and some forms of EDMD and ALS, might benefit from new strategy
posted on April 18, 2009 - 9:00pm
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.
New strategy may lessen muscle damage and improve function in DMD
posted on April 13, 2009 - 5:00pm
Investigators conducting experiments in mice with a disease resembling Duchenne muscular dystrophy (DMD) believe they’ve uncovered a new strategy to protect against muscle damage and improve strength in this disease.
Andrew Marks at Columbia University in New York coordinated the team, which included researchers from Montpellier (France) University and other institutions in Montpellier. They...
Direct injections of utrophin protein into dystrophin-deficient mice strengthened their muscles
posted on May 27, 2009 - 9:00pm
MDA grantee James Ervasti and colleagues at the University of Minnesota-Twin Cities in Minneapolis have found that a protein known as utrophin, injected into mice lacking the dystrophin protein and showing a disease resembling Duchenne muscular dystrophy (DMD), conferred significant benefits.
The experiments Ervasti and colleagues describe online May 26, 2009, in PLoS Medicine, are the first to...
Blocking a protein associated with inflammation and scarring helped mice with a DMD-like disease
posted on May 22, 2009 - 4:23pm
A protein called osteopontin has been implicated as a cause of some of the detrimental inflammation and scarring ("fibrosis") of muscle tissue that takes place in Duchenne muscular dystrophy (DMD). Eliminating osteopontin was beneficial to mice with a DMD-like disease, and the researchers concluded that reducing osteopontin should be investigated as a possible therapy for DMD.
posted on January 31, 2009 - 10:00pm
On Jan. 21, AVI BioPharma of Portland, Ore., announced its experimental compound AVI4658 for the treatment of Duchenne muscular dystrophy (DMD) yielded promising results in a phase 1 clinical trial in the United Kingdom.
