Investigators have safely injected gamma-sarcoglycan genes into an arm muscle in nine people with limb-girdle muscular dystrophy type 2C; high-dose recipients produced gamma-sarcoglycan protein
A March 2012 podcast from Nationwide Children's Hospital in Columbus, Ohio, presents the results of a phase 1 trial of gene therapy for gamma-sarcoglycan-deficient limb-girdle muscular dystrophy (LGMD), also known as LGMD2C.
The pace of research can seem unreasonably slow; here are a few reasons why
John Porter from the National Institutes of Health likes to start talks by noting, “It’s a great time to be a mouse with a neuromuscular disease.” Exciting research results are regularly reported, where a treatment appears to cure one neuromuscular disease or another in a mouse — yet there are few treatments available today for people with any of these diseases, and only a few treatments in human...
Researchers at six centers are studying possible cardiac benefits of dietary supplement coenzyme Q10 and cardiovascular drug lisinopril in three MD forms
Researchers at five U.S. and one Canadian center are conducting a clinical trial of the medications coenzyme Q10 and lisinopril to determine their possible beneficial effects on heart function in Duchenne muscular dystrophy (DMD), Becker muscular dystrophy (BMD) and five forms of limb-girdle muscular dystrophy (LGMD).
Research items about Friedreich's ataxia, myasthenia gravis, mitochondrial myopathies, type 1 myotonic dystrophy, gene therapy and gene modification
Edison drugs target FA, mitochondrial diseases
The biggest problem at an ER may not be the one you go in with, but the one you encounter there
When a medical emergency strikes — and the patient is a person with a neuromuscular disease — it’s not just getting to the emergency room quickly that’s critical. It’s also critical to ensure the ER staff understands the patient’s special needs caused by muscle disease.
Reports from the American Academy of Neurology annual meeting about Duchenne, limb-girdle and myotonic muscular dystrophies, dermatomyositis, myasthenia gravis and spinal muscular atrophy
Below are brief reports and links to more information about neuromuscular disease research presented at the 63rd annual meeting of the American Academy of Neurology (AAN), held in Honolulu April 9-16, 2011.
A change in the LTBP4 protein gene reduces muscle-damaging signals in mice with a disease resembling LGMD2C
New research has shown that a change in a gene not previously connected to type 2C limb-girdle muscular dystrophy (LGMD2C) modifies the severity of the disease in mice and is likely to do the same in people with this and perhaps with related types of muscular dystrophy.