Muscular Dystrophies

'Gapmer Antisense' Stops Disease in MMD1 Mice

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.

Researchers Studying Children's Use of Mobility Devices

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.

PTC Update on the Development of Ataluren in Nonsense-Mutation DMD/BMD

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

EDMD, LGMD1B: Two More Leads for Treating Cardiomyopathy

Update (Sept. 10, 2012): Information about the potential for serious side effects with temsirolimus and rapamycin was added to this article.

Encouraging Preclinical Results in Membrane Sealant Drug for DMD

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).

A Closer Look: Extended Eteplirsen Treatment Benefits Walking in DMD

Editor's note (July 30, 2012): This story was revised to include information about the specific mutations being targeted by eteplirsen.

Exon-Skipping Trial Results Are a 'Major Advance' in DMD Treatment

Editor's note (July 24, 2012): For a more in-depth discussion of the exon-skipping trial results, see A Closer Look: Extended Eteplirsen Treatment Benefits Walking in DMD.

Research Briefs: LGMD, Myofibrillar Myopathy

Update (Aug. 8, 2012):This story was updated to reflect the availability of a podcast on the dysferlin gene transfer study.

Zebrafish research models mimic myofibrillar myopathy

MMD Briefs: New Drug Development, Perceptions of Modafinil

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.

MD Briefs: Corrected Stem Cells, Membrane Sealants

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.

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