MDA's 2015 Scientific Conference points the way toward tomorrow's therapies
Last month, MDA brought together more than 400 of the nation’s leading scientists, researchers and professionals specializing in neuromuscular disease research and care at its 2015 Scientific Conference in Washington, D.C., March 11–14. This pre-eminent gathering, hosted every other year, was designed to accelerate discoveries and drug development for families fighting neuromuscular diseases.
Research updates and clinical trials information
What About My Disease?
Readers sometimes wonder what’s happening with research for their diseases when they don’t see news about them for a while in the pages of Quest. But keep in mind: Research that seems to be for one disorder often has spillover implications for others.
An MDA-supported research team has found in animal experiments that plasmapheresis can reduce the number of unwanted antibodies to gene therapy delivery vehicles
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
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
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
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
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.