Rare Disease Report

CEO of Abeona Discusses Gene Therapy for Sanfilippo Syndrome

FEBRUARY 25, 2017
Tim Miller, PhD

At WORLDSymposium in San Diego, CA, we talked with Tim Miller, PhD, CEO of Abeona Therapeutics about the gene therapy study they are conducting in patients with sanfilippo syndrome. 


Flanigan KM, Truxal KB, McBride KL, et al. Systemic gene transfer of scAAV9.U1a.hSGSH for MPS IIIA: tolerability and preliminary evidence for a biochemical effect. Presented at WORLDSymposium; San Diego, CA; February 13-17, 2016.

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