Awarded Grants

LGMD2I zebrafish characterization and drug screening

Dr. Louis Kunkel

Boston Children’s Hospital (Boston, MA)

Biomarkers discovery in LGMD2I

Drs. Sebahattin Cirak and Yetrib Hathout

Children’s National Medical Center (Washington, DC)

Stem Cell therapy in mouse model of LGMD2I

Drs. Casey Childers & David Mack

University of Washington (Seattle, WA)

Assessment of Muscle Function in mouse model of LGMD2I

Dr. Dan Rodgers

Washington State University (Pullman, WA)

Characterization of FKRP mutant mouse model

Dr. Kanneboyina Nagaraju

Children’s National Medical Center (Washington, DC)

Genetic correction of patient-specific LGMD2I iPS cells and the regenerative potential of their skeletal muscle derivatives

Dr. Rita Perlingeiro

University of Minnesota (Minneapolis, MN)

Discovery of chemical chaperones for the treatment of FKRP-related limb girdle muscular dystrophy 2I

Dr. Sebahattin Cirak

Children’s National Medical Center (Washington, DC)

Development and characterization of novel zebrafish models of dystroglycanopathies

Dr. James Dowling

University of Michigan (Ann Arbor, MI)

Generation of a monoclonal antibody against FKRP

Dr. Isabelle Richard

Généthon (Evry, France)

Generation and characterization of antibodies to alpha-dystroglycan

Dr. Susan Brown

Royal Veterinary College (London UK)

Dr. Glenn Morris

RJAH Orthopaedic Hospital Trust (Oswestry, UK)

Development of dystroglycanopathy patient specific cell based assays for drug screening

Dr. Anne Bang

Sanford-Burnham Medical Research Institute (La Jolla, CA, USA)

Development of Soluble mini-Agrin as a Treatment for Dystroglycanopathies

Dr. Jasbir Seehra

Achelois Biosciences (Lexington, MA, USA)