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.
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).
Longtime MDA research grantee honored for work in LGMD gene therapy
A team headed by neurologist Jerry Mendell, a longtime MDA research grantee and director of the MDA Clinic at Nationwide Children's Hospital in Columbus, Ohio, has received the prestigious Annals of Neurology prize for an outstanding contribution to clinical neuroscience.
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.
Five of six people with limb-girdle muscular dystrophy type 2D produced protein from the new genes
Results from an MDA-supported, phase 1 study of gene therapy for the type 2D form of limb-girdle muscular dystrophy (LGMD2D) show sustained protein production from the transferred genes in two out of three trial participants six months after injection of the genes into a foot muscle.
The new results follow an announcement last year showing protein production from transferred genes in the first...