The Samantha Brazzo Foundation, the LGMD2I Research Fund and Cure CMD will jointly fund a research project to support the development and characterization of a stable FKRP zebrafish model to be used for screening of potential drug candidates.
Candidate drugs will also be tested in another zebrafish model of dystroglycanopathy (Pomt2 deficiency) in parallel to the FKRP model, for comparison. This will bring further insights into the disease process among the alpha-dystroglycan-related diseases. In order to potentially expand the impact of the discoveries made in this project, all results will be shared through an open-source database.
The project will be led by Dr James Dowling (University of Michigan), who has proven track-record in generating stable zebrafish models for other muscle diseases. The stable zebrafish model of FKRP mutations is a key tool for identifying new therapies for LGMD2I, and by screening drugs in two dystroglycanopathy models, we hope to maximize the advancements in drug discovery for this group of disorders.