Cure CMD Funds 3 New Research Grants in 2014
- 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 serum.
- Micro RNA Biomarkers in LAMA2-CMD- Prof. Francesco Muntoni. This study will look at micro RNA levels in the blood in children and adults with LAMA2-CMD. Micro RNA are small pieces of RNA (genetic language) that can be found in both muscle and blood. Prof Muntoni has recently completed a study looking at the profile of micro RNA in boys with Duchenne muscular dystrophy and in people with Collagen VI related myopathy. The goal will be to determine if people with LAMA2-CMD have a distinct profile that can be used in clinical trials to measure if a drug is having an impact.
- ISPD Function and Drug Screen- Dr. Sebahattin Cirak. This study will develop synthetic ISPD protein: both with normal function and with altered function (similar to what is found in patients). ISPD is a gene when mutated leads to a dystroglycanopathy. Dr. Cirak will investigate the function of ISPD and perform a drug screen to look for compounds that can stabilize the ISPD protein and improve its function even when altered
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