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Late Breaking News

Abeona Therapeutics announce enrollment in its Transpher A and Transpher B studies.

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Abeona Therapeutics Reports Top-Line Data from Phase 1/2 Gene Therapy Trial in MPS IIIA

Abeona Therapeutics Reports Top-Line Data from Phase 1/2 Gene Therapy Trial in MPS IIIA • ABO-102 results presented at WORLDSymposium for Lysosomal Diseases show significant time- and dose-dependent reduction of underlying disease pathology, including decreased CSF and urine GAGs (HS fragments) and diminished liver volumes • Evidence of cognitive benefit at six months post treatment in Cohort...

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Abeona Therapeutics Reports Top-Line Data from Phase 1/2 Gene Therapy Trial in MPS IIIA

Abeona Therapeutics Reports Top-Line Data from Phase 1/2 Gene Therapy Trial in MPS IIIA • ABO-102 results presented at WORLDSymposium for Lysosomal Diseases show significant time- and dose-dependent reduction of underlying disease pathology, including decreased CSF and urine GAGs (HS fragments) and diminished liver volumes • Evidence of cognitive benefit at six months post treatment in Cohort...

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Upcoming Events

Stay tuned for our 2019 fundraising efforts and events. 

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Kirby Wilson

Brad and I formed this Foundation because of our four-year-old daughter, Kirby, the disease she was afflicted with, and our hope that Sanfilippo and its devastating progression would not manifest itself within her — our beautiful bundle of joy. The solution seemed simple. Raise funds to enable researchers to advance and expand their work to find a cure. We chose to fight this disease. Now 19...

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