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
Sio Gene Therapies Announces First Patient Dosed in Clinical Trial of AXO-AAV-GM2 in Patients with Tay-Sachs and Sandhoff Disease
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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.