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

Clinical trial of molecular therapy for muscular dystrophy yields significant positive results

CHAPEL HILL, N.C. – A molecular technique originally developed at the University of North Carolina at Chapel Hill has taken one step closer to becoming a treatment for the devastating genetic disease Duchenne muscular dystrophy.

Read more

Human protein improves muscle function of muscular dystrophy mice

PROVIDENCE, R.I. [Brown University] — A novel potential therapy based on a natural human protein significantly slows muscle damage and improves function in mice who have the same genetic mutation as boys with the most common form of muscular dystrophy, according to a paper published online Dec. 27 in the Proceedings of the National Academy of Sciences.

Read more

New mouse model for duchenne muscular dystrophy implicates stem cells, Stanford researchers say

STANFORD, Calif. — For years, scientists have tried to understand why children with Duchenne muscular dystrophy experience severe muscle wasting and eventual death. After all, laboratory mice with the same mutation that causes the disease in humans display only a slight weakness. Now research by scientists at the Stanford University School of Medicine, and a new animal model of the disease they developed, points a finger squarely at the inability of human muscle stem cells to keep up with the ongoing damage caused by the disorder.

Read more

First clinical trial of gene therapy for muscular dystrophy lends insight into the disease

CHAPEL HILL – A clinical trial designed to replace the genetic defect causing the most common form of muscular dystrophy has uncovered an unexpected aspect of the disease. The trial, based on therapy designed by scientists at the University of North Carolina at Chapel Hill School of Medicine, showed that some patients mount an immune response to the dystrophin protein even before they have received the gene therapy.

Read more

Gene therapy reveals unexpected immunity to dystrophin in patients with Duchenne muscular dystrophy

Natural immunity to dystrophin may contribute to muscle disease and complicate experimental therapies

An immune reaction to dystrophin, the muscle protein that is defective in patients with Duchenne muscular dystrophy, may pose a new challenge to strengthening muscles of patients with this disease, suggests a new study appearing in the October 7, 2010, issue of The New England Journal of Medicine.

Read more

Rochester leads international effort to improve muscular dystrophy treatment

A large international study aimed at improving the care of muscular dystrophy patients worldwide is being launched by physicians, physical therapists, and researchers at the University of Rochester Medical Center.

Read more

Discovery opens door to therapeutic development for FSH muscular dystrophy

Scientists are closer to understanding what triggers muscle damage in one of the most common forms of muscular dystrophy, called facioscapulohumeral muscular dystrophy (FSHD).

FSHD affects about 1 in 20,000 people, and is named for progressive weakness and wasting of muscles in the face, shoulders and upper arms. Although not life-threatening, the disease is disabling. The facial weakness in FSHD, for example, often leads to problems with chewing and speaking.

Read more

Heart drug effective for treating symptom of muscular dystrophy

Contact: Tom Rickey
tom_rickey@urmc.rochester.edu
585-275-7954
University of Rochester Medical Center
A medication most often used to treat heart arrhythmias also reduces a central symptom of myotonic dystrophy, the most common type of muscular dystrophy in adults.

Read more

Next Page »