Ben Philpot, PhD Partners with Deerfield & Pinnacle Hill

October 21, 2024

Deerfield and Pinnacle Hill have partnered with Dr, Ben Philpot, UNC Chapel Hill, to advance a new therapeutic for AS! Their work focuses on manipulating gene expression and this collaboration will accelerate work previously funded by the ASF to the next phase of development.

Rugonersen Phase 1 TANGELO Study Results Published in Nature Medicine

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Ultragenyx Pharmaceutical Receives Breakthrough Therapy Designation from FDA for GTX-102

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First Patient(s) Dosed in Ionis Phase 3 ION582 Study

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MavriX Bio Announces New Potential Gene Therapy for Angelman Syndrome

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The mission of Angelman Syndrome Foundation is to advance the awareness and treatment of Angelman syndrome through education and information, research and support for individuals with Angelman syndrome, their families and other concerned parties. We exist to give all of them a reason to smile, with the ultimate goal of finding a cure.

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