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

Abeona Therapeutics Announces Fast Track Designation from FDA for ABO-102 in Sanfilippo Syndrome Type A

The FDA has granted Abeona Therapeutics Fast Track status to the Gene Therapy trial to treat Sanfilippo Syndrome Type IIIA. ... Learn more

Abeona Therapeutics Provides Update from ABO-102 Phase 1/2 MPS IIIA Clinical Trial at Orphan Drugs & Rare Disease Conference

Exciting 30-day trial data has been released by Abeona Therapeutics. Highlights include Gene Therapy has been well-tolerated in low-dose Cohort of ... Learn more

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