Stem Cell Gene Therapy Treatment for Cystinosis Potential Treatment for Alzheimer’s Disease

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In 2003, the mission of the Cystinosis Research Foundation was to find better treatments and a cure for cystinosis. Since then, the research funded by CRF has resulted in significant advances in the field of cystinosis have been accomplished. This research has impacted other more prevalent diseases and disorders, including Danon disease and Friedreich’s Ataxia. […]

Stem Cell Trial Phase I/II Updates by Stéphanie Cherqui, PhD

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Dr. Stéphanie Cherqui provided an update on the stem cell gene therapy trial at the 2022 Day of Hope Family Conference. Five patients have now received the transplant, and she has good follow-up data on two of them. White blood cell cystine levels in these patients have gone down to normal despite not being on […]

The Third Patient Was Transplanted November 16, 2020!

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From Rocky Seachord, Jacob’s Mom: “He is in very good spirits and I think it is a good omen that he is in the very same room that Jordan was in… The transplant, considered Day 0, is Monday the 16th. This will be his new birthday. I can’t even imagine the possibilities in his life […]