ADVANCE 2024: Advances in Preclinical Research For Sanfilippo

Advances in Preclinical Research For Sanfilippo

Topic: Peripheral Neural Stem Cell Models and Drug Discovery for Sanfilippo Syndrome | Ryan J. Weiss, PhD and Nadja Zeltner, PhD

Topic: AVP6, a promising small molecule for treatment of Sanfilippo disease | Travis Moore, Msc, PhD candidate and Patricia Dubot, MD, PhD

Topic: Development of gene therapy for MPS IIIC | Steven Gray, PhD

Q&A with presenters

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