MFRF has provided leadership and raised approximately $2.5 million of funding for projects spanning its mission and vision across three pillars: Research of Treatments and Cures, Education and Genetic Screening, and care through Nursing School Educational programs.
MFRF has celebrated wonderful progress in education, genetic testing, and gene therapy patient trials for many affected children and families with fatal genetic diseases of the brain
Amicus Therapeutics
Brain Institute College of Medicine and College of Nursing
Jewish Genetic Disease Consortium
National Organization of Rare Disorders (NORD)
Family Services, Quality Control, Research Initiative
College of Nursing and Wellness Screenings
Genzyme Genetics (Testing and Counseling) & Genzyme Therapeutics
Scripps Research Institute
Sio Gene Therapies
College of Medicine
Center for Medical Genetics
Genetic Testing and Counseling
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MFRF has celebrated
wonderful progress in gene therapy patient trials, education, and genetic testing for many affected children and families with fatal genetic diseases of the brain. Still, there is so much more to do, including earlier detection and expanding treatment availability.