Future Gene Therapy Trial

I had the opportunity to sit in on a conference call about Sanfilippo Syndrome and a potential gene therapy trial in the US. This is very exciting news for our community. Families have been working for many long years to raise money to further research. This particular doctor and her staff are moving forward with a Natural History Study for patients with MPS III A & B. Hopefully within the next 1.5 - 2 years they will begin a small patient trial to test the safety and efficacy of the treatment. The mouse models are showing very positive results and we hope that translate to similar results in children. Unfortunately, it is too late for Waverly & Oliver. However they have done their part and will continue to do so, to further research. With their help, I am confident a treatment will one day be available for families.


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