It’s with great optimism that we share exciting news!
With the backing of research that this tournament and all its supporters helped fund at UNC Children’s Hospital, NeuroGT, a clinical-stage gene therapy company, has received FDA approval of their Investigational New Drug (IND) application for an AAV-9 gene therapy treatment of Sanfilippo Syndrome Type B. A combined Phase I/II clinical trial is planned to begin in early 2027. Research continues on gene therapy treatments for other types of Sanfilippo and MPS disorders.
We are forever grateful for your support in working toward bringing life changing therapies to our Sanfilippo and MPS children and their families.
We look forward to sharing additional updates and seeing you at our 11th Annual Tournament.
