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Late Breaking News

Late Breaking News

The good news continues with Abeona Therapeutics and Nationwide Children's Hospital

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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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Purple Lemonade Stand

Purple Lemonade Stand

Along with other family foundations involved in raising funds toward the cure for Sanfilippo Syndrome, stuff we have been invited to participate in a national effort called Purple Lemonade – A Stand for Sanfilippo Syndrome. It was the vision of Grey Chapin, a six-year-old determined to fund a cure for the disorder that afflicts her older sister Blair. Please take a moment to read further to...

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Webinar: October 8

Webinar: October 8

Nationwide will be hosting a webinar October 8th from 2:00 – 3:30 EST to further explain the MPS IIIA/IIIB Natural History Study, including the rationale, aims, and plans for enrollment. This will be a 90-minute webinar with 45 minutes of slides/presentations followed by 45 minutes of questions. Further details to follow, including call-in information. Space is limited to 250 attendees. Learn...

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