A recent article highlighted a possible treatment for people with DNM2 mutations and centronuclear myopathy. The medication is called mestinon (pyridostigmine). It is a medication that works at the neuromuscular junction, where the nerve meets the muscle. Click here to view/download the article. The reason people with DNM2 mutations get a muscle condition is still [...]
Losartan Trial in Dyw Mice- Dr. Sweta Girgenrath. This drug trial in the LAMA2-CMD mouse model will look at whether early treatment (two weeks) and late treatment (4 weeks) confers the same benefit and what the benefit of sustained treatment is. This is a two year grant that will evaluate biomarkers in mouse muscle and [...]
June 25, 2013 — Researchers at Boston University College of Health & Rehabilitation Sciences: Sargent College have identified a combinatorial therapeutic approach that has proven effective in treating muscular dystrophy in a mouse model. The findings, published in Human Molecular Genetics, represent a paradigm shift for the treatment of muscular dystrophy as well as a [...]
Also posted in Press Releases
From Muscular Dystrophy Campaign A group of scientists from Italy has used exon skipping technology to restore the production of a protein called collagen VI in a model system of Ullrich congenital muscular dystrophy. The researchers used cells grown in the laboratory to demonstrate proof of principle that exon skipping might have the potential to [...]
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Investigators at the University of Michigan in Ann Arbor are conducting a study of the progression of centronuclear myopathies (CNMs), including the type known as myotubular myopathy (MTM), central core disease (CCD) and multiminicore disease. The study is intended to: • assess the progression of CNMs, CCD and multiminicore disease • identify potential therapeutic targets for these disorders • help [...]
Check out this publication by Resmed that describes techniques on how to make the introduction of respiratory support for your little one less threatening.
Cure CMD, the LGMD2I Fund and the Newcastle TREAT-NMD office announce a collaboration to promote clinical trial readiness for LGMD2I. In anticipation of an LGMD2I clinical trial, we are working to align the 2 existing registries, one specific for FKRP and one for each of the other CMD-LGMD alpha dystroglycan related dystrophy (aDG-RD, dystroglycanopathy) genes. [...]
2012 is starting off with a bang- $14,910 raised in response to a $10,000 challenge by Cure CMD’s Chair, Anne Rutkowski, nets $25,000 for research! Consider setting up a monthly donation through our donation page to help us reach our 2012 goal to raise $500,000!!
Cure CMD 2012 Funding: CMDIR Project Manager, the Myomatrix 2012 Scientific Conference, an LMNA-CMD iPS cell line and CMD Comparative Outcome Measure Study (CMD COM-Year 3).