Mission: There is no cure or treatment strategy for patients with FSH Muscular Dystrophy (FSHD). This debilitating disease slowly consumes skeletal muscle, robbing people of the active, healthy, and independent years of their lives. For over a decade, Friends of FSH Research (Friends) has supported research studies that have contributed to our understanding of FSHD, offering the hope of treatment to the over 500,000 people living with the disease. Friends was started by the family and friends of Terry and Rick Colella, whose son has FSHD. The goal of the organization was to stimulate research on FSHD in the Pacific Northwest. By reaching out to investigators at the University of Washington, Seattle Children’s Hospital, and the Fred Hutchinson Cancer Research Center, we were able to provide seed monies for projects that would ultimately attract over $15 million dollars in funding. Although there now exists an impressive number of researchers supporting FSH research, there are even more projects that need to be funded in order to accelerate the goal to develop a treatment or cure.
Results: * Scientists have reached a consensus on the root cause of FSHD and have identified two molecular targets for intervening with the disease. * Platforms have been developed for lead compound identification and scientists are now performing massive experiments to look for compounds that interfere with the disease. * Other strategies, including viral gene therapy, are also showing great promise. * Preclinical models of the disease have been developed, and are being used to test lead compounds and strategies. * Clinical trial endpoints are being established so we know, when we find a therapy, whether it will work or not.
Target demographics: accelerate a treatment or cure for FSH Muscular Dystrophy
Direct beneficiaries per year: 3+ researchers
Geographic areas served: Seattle, Washington
Programs: The mission of Friends of FSH Research is to accelerate the discoveries that will lead to treatments or cures for FSHD. To achieve our goal, we actively manage a portfolio of research that supports a pipeline for drug discovery. Our board, staff, volunteers, and world-class scientists are fully committed to transparent, reproducible, and transformative science that will inspire hope for people with FSHD.