Muscular Dystrophies

An experimental treatment for type 1 myotonic muscular dystrophy (MMD1, or DM1) has corrected several aspects of the disease in an MMD1 mouse model.

A year after treatment with an experimental antisense oligonucleotide dubbed ASO 445236 ended, positive effects remained apparent in the mice.

If you're the parent of a young child who uses a power mobility device, researchers at the University of British Columbia (UBC) and Sunny Hill Health Centre for Children — both in western Canada — are interested in hearing from you. The study is open to U.S. residents.

New Jersey biopharmaceutical company PTC Therapeutics recently declared its intention to do whatever it takes — including an additional clinical trial — to seek approval for ataluren (formerly PTC124) as a treatment for Duchenne muscular dystrophy (DMD) and Becker muscular dystrophy (BMD) caused by non

Phrixus Pharmaceuticals in Ann Arbor, Mich., reported July 19, 2012, that its experimental drug Carmeseal demonstrated a beneficial effect on the diaphragm (the primary breathing muscle) in mice with a disease resembling Duchenne muscular dystrophy (DMD).

Zebrafish research models mimic myofibrillar myopathy

Type 1 myotonic dystrophy (MMD1, or DM1) and type 2 myotonic dystrophy (MMD2, or DM2) are complex, multisystem disorders caused by similar genetic flaws on chromosome 19 (MMD1) and chromosome 3 (MMD2). Treatments that target the underlying molecular causes of MMD1 and MMD2 are in development.

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.