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Helping Find a Cure for Sanfilippo Syndrome

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  • About Sanfilippo Syndrome

    Sanfilippo Syndrome, or Mucopolysaccharidosis IIIA (MPS-IIIA) is a genetic lysosomal storage disease (LSD) caused by the body’s inability to produce the enzyme Heparan N-sulfatase. The body uses the ... Learn more

    About Sanfilippo Syndrome

About the Abby Grace Foundation

About Us

The Abby Grace Foundation remains HOPEFUL for a CURE for Sanfilippo Syndrome. There is currently no cure or even a treatment for this always fatal disease.

There is exciting research on the horizon for children born with Sanfilippo Syndrome. A Clinical Trial treating a small number of children began at Nationwide Children's Hospital in Ohio in the spring of 2016. The Abby Grace Foundation, along with many other family-formed foundations, helped support the funding of the Gene Therapy Clinical Trial.

While we must wait for results of the trial, we overwhelmingly feel a sense of urgency to support other potential research.  We will continue fighting to cure Sanfilippo Syndrome for our daughter Abby Grace and the many other children born with this devastating disease.

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Recent News

Gene Therapy Trial Shows Encouraging Early Biopotency Signals

We are extremely encouraged to receive this early data from the Gene Therapy Trial. Abeona Therapeutics provides update on initial subjects in ... Learn more

Abeona Therapeutics Doses First Sanfilippo Patient With Gene Therapy

Exciting news to share! Abeona Therapeutics doses the first patient in Phase 1/2 Trial with ABO-102 Gene Therapy for patients with Sanfilippo Syndrome ... Learn more

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