The LGMD2I Research Fund, the Samantha Brazzo Foundation and Cure CMD will jointly fund a research project to support drug screening on mutant forms of FKRP, in the search for compounds that restore normal protein function. The project, led by Dr. Sebahattin Cirak at the Children’s National Medical Center, will use a novel approach for the analysis of FKRP mutants and will allow for the identification of highly selective drug candidates that could be further tested in patients’ cells and in murine models of FKRP deficiency. This project will help better understand the function of FKRP and could potentially lead to the discovery of therapeutic drugs against LGMD2I.
New grant to study mutant forms of FKRP
April 2, 2013