Cedars-Sinai research: Preclinical muscular dystrophy data shows promise

Cedars-Sinai Heart Institute researchers have found that an experimental compound may help stem the debilitating effects of muscular dystrophy by restoring normal blood flow to muscles affected by the genetic disorder.

Read more

An important breakthrough in the fight against muscular dystrophies

 

An important breakthrough could help in the fight against myotonic dystrophy. The discovery, recently published in the prestigious scientific journal Cell, results from an international collaboration between researchers at the IRCM, the Massachusetts Institute of Technology (MIT), the University of Southern California and Illumina. Their findings could lead to a better understanding of the causes of this disease.

Read more

Real-life spider men using protein found in venom to develop muscular dystrophy treatment

 

While Spider-Man is capturing the imagination of theatergoers, real-life spider men in Upstate New York are working intently to save a young boy’s life.

It all began in 2009, when Jeff Harvey, a stockbroker from the Buffalo suburbs, discovered that his grandson, JB, had Duchenne muscular dystrophy. The disease is fatal. It strikes only boys, causing their muscles to waste away.

Read more

New gene transfer strategy shows promise for limb girdle and other muscular dystrophies

The challenge of treating patients with genetic disorders in which a single mutated gene is simply too large to be replaced using traditional gene therapy techniques may soon be a thing of the past. A Nationwide Children’s Hospital study describes a new gene therapy approach capable of delivering full-length versions of large genes and improving skeletal muscle function. The strategy may hold new hope for treating dysferlinopathies and other muscular dystrophies.

Read more

New compound holds promise for treating Duchenne MD, other inherited diseases

 

Scientists at UCLA have identified a new compound that could treat certain types of genetic disorders in muscles. It is a big first step in what they hope will lead to human clinical trials for Duchenne muscular dystrophy.

Read more

Clinical trial first to test heart drug regimen for Duchenne muscular dystrophy

The first landmark randomized clinical trial for a cardiac drug regimen in Duchenne muscular dystrophy (DMD) is testing whether earlier treatment can stop or slow down heart damage that usually kills people with the disease.

Read more

U of M researchers develop new muscular dystrophy treatment approach using human stem cells

Researchers from the University of Minnesota’s Lillehei Heart Institute have effectively treated muscular dystrophy in mice using human stem cells derived from a new process that – for the first time – makes the production of human muscle cells from stem cells efficient and effective.

Read more

Next-generation DNA sequencing to improve diagnosis for muscular dystrophy

Scientists at The University of Nottingham have used a revolutionary new DNA-reading technology for a research project that could lead to correct genetic diagnosis for muscle-wasting diseases.

The technique could be used to offer people with muscular dystrophy, or a related neuromuscular condition, a more accurate prognosis, which would enable them to make more informed choices on life decisions, including family planning.

Read more

Study reveals enzyme function, could help find muscular dystrophy therapies

Researchers at the University of Iowa have worked out the exact function of an enzyme that is critical for normal muscle structure and is involved in several muscular dystrophies. The findings, which were published Jan. 6 in the journal Science, could be used to develop rapid, large-scale testing of potential muscular dystrophy therapies.

Read more

Clinical trial for muscular dystrophy demonstrates safety of customized gene therapy

Researchers at the University of North Carolina at Chapel Hill have shown that it is safe to cut and paste together different viruses in an effort to create the ultimate vehicle for gene therapy. In a phase I clinical trial, the investigators found no side effects from using a “chimeric” virus to deliver replacement genes for an essential muscle protein in patients with muscular dystrophy.

Read more

Next Page »