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A Wish to live…
A Mission to find a Cure for Sanfilippo
In February 2015, our daughter Aislinn, was diagnosed with the rare genetic disorder Sanfilippo Syndrome, MPSIIIA. With no available treatment or cure, Sanfilippo stole her voice, her mobility, her ability to eat and ultimately, in October 2022, it stole her tomorrows.
Aislinn’s life continues to be honored through Aislinn’s Wish Foundation, which seeks to find a cure for Sanfilippo and other MPS Disorders.
FOREVER IN OUR HEARTS
It’s with great optimism that we share exciting news!
With the backing of research that Aislinn’s Wish 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.
UPCOMING EVENTS