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
Family Driven Development of Treatments for Rare Pediatric Neurological Diseases
Published article in the prestigious Clinical Pharmacology and Therapeutics ...
Read More The Year in Review summary 2021
The Year in Review summary 12/16/21. https://conta.cc/31S4v9J
Read More Sio Gene Therapies Announces Positive Interim Safety and Biomarker Data from Ongoing Phase
"Sio Gene Therapies Announces Positive Interim Safety and Biomarker ...
Read More Sio Gene Therapies Announces First Patient Dosed in Clinical Trial of AXO-AAV-GM2 in Patients with Tay-Sachs and Sandhoff Disease
Sio Gene Therapies Announces First Patient Dosed in Clinical ...
Read More Taysha Gene Therapies “A Clinical Trial Option for Children with Tay-Sachs Disease or Sandhoff”
Taysha Gene Therapies "A Clinical Trial Option for Children ...
Read More Axovant Gene Therapies Announces FDA Clearance of IND for AXO-AAV-GM2 Gene Therapy in Tay-Sachs and Sandhoff Diseases
AXO-AAV-GM2 is the first investigational gene therapy to receive ...
Read More TEAM DAD Parenting Series: Dad Highlights Program
TEAM DAD Parenting Series: Dad Highlights Program GAME ON ...
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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.
