General Research

An MDA-supported research team has found in animal experiments that plasmapheresis can reduce the number of unwanted antibodies to gene therapy delivery vehicles

posted on December 6, 2013 - 2:45pm
A research group supported in part by MDA has found that a blood-filtering strategy known as plasmapheresis looks promising as a way to help overcome one type of unwanted immune response to gene transfer therapy. Gene transfer therapy is the addition of therapeutic genes to treat disease, and it is often administered via transport vehicles made from viruses. Louis Chicoine at Nationwide Children'...

Investigators conducting an online survey to gather information from people with inclusion-body myositis report early results and ask a few more questions

posted on November 27, 2013 - 5:00am
Investigators conducting an online survey launched in January 2013 to gather anonymous information from people with inclusion-body myositis (IBM) are now reporting preliminary results and are asking the original respondents to complete a short supplementary survey.

A gene therapy method tested in human cells ‘significantly’ increased production of frataxin, the protein deficient in Friedreich's ataxia

posted on August 8, 2012 - 6:00am
Human cells treated with engineered transcription activation-like effector (TALE) proteins produced two to three times more frataxin protein than did control cells, a team of researchers has reported.

A new research mouse may speed development of treatments for McArdle disease, and a conference this August will focus on several glycogen storage diseases

posted on July 5, 2012 - 6:00am
New research mouse mimics McArdle disease Scientists in Spain have developed mice with a disorder that closely resembles the human metabolic muscle disorder known as McArdle disease (also called phosphorylase deficiency, myophosphorylase deficiency and glycogenosis type 5). The mice are expected to speed testing of treatments for this disorder.

Genetically corrected human stem cells help mice with a disease resembling limb-girdle MD; cell membrane sealants are being studied in Duchenne and other forms of muscular dystrophy

posted on July 3, 2012 - 11:49am
LGMD2D mice benefit from corrected human stem cells A multinational team of scientists successfully transplanted genetically corrected muscle stem cells derived from people with type 2D limb-girdle muscular dystrophy (LGMD2D) into LGMD2D research mice and saw better muscle function in these mice than in similar mice that didn't receive the cells.

Researchers are pursuing several strategies to identify or modify stem cells that can be used to repair damaged muscle fibers

posted on June 14, 2012 - 10:21am
Update (July 10, 2012) — This story was updated to reflect the availability of a podcast with MDA research grantee Emanuela Gussoni, who discusses the development of stem-cell-based treatments for muscular dystrophies.

Top researchers are discussing notable developments in neuroscience, including research in many neuromuscular diseases, at the annual American Academy of Neurology meeting April 21-28

posted on April 24, 2012 - 4:00am
Top scientists and clinicians from around the world are discussing the latest research in neuroscience, and the care of individuals with nerve and muscle diseases, at the 2012 annual meeting of the American Academy of Neurology in New Orleans, April 21-28.

In this first of several reports on MDA's Clinical Conference, the role of genetics and immunology in different neuromuscular diseases is described

posted on March 19, 2012 - 3:08pm
More than 500 physicians, allied health care professionals and MDA staff attended the MDA's 2012 Clinical Conference in Las Vegas, March 4-7. The program emphasized: