MDBR - Congenital Muscular Dystrophy

One $41,831 grant available. CMD are a group of genetically inherited multi-system disorder with symptom onset at birth and progressive muscle weakness, respiratory insufficiency, contractures and scoliosis.  Failure to thrive, cardiac arrhythmias, subclinical cardiomyopathy and seizures may complicate disease course. The purpose of this RFA is to promote discovery of underlying disease mechanisms and preclinical development of potential therapies, as well as the clinical translation of those efforts.  Areas of interest include, but are not limited to, understanding the cause of disease, unraveling pathways involved in disease, identifying novel drug targets or gene therapies, and testing new strategies to treat disease or any of its incapacitating consequences (e.g. contractures).  In addition, applications may propose to create or improve disease models (e.g. animal models, patient-derived cell models), biomarkers or functional outcome measures to asses therapeutic impact. Priority will be given to research projects targeting Collagen VI (Ullrich).  This grant is made possible by Team Cure CMD and the Cure CMD organization.


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