Wow, what a year 2022 has been so far! I have a wonderful boyfriend in my life (we are getting a puppy soon), the beloved show, This is Us is ending and I was the 5th patient to receive a stem cell transplant! For those of you who don’t know the show, This is Us, the show’s message …
Natalie Stack 5th Patient Stem Cell and Gene Therapy Trial – March 29, 2022 Read More »
By Tyler Joynt, San Diego, California As a kid, I really only knew three things about cystinosis. Doctors, sickness, and the San Diego Zoo. From an early age I remember throwing up often, going to the ER regularly, and seeing doctors frequently. I was lucky enough to be diagnosed before I was two years old, …
Tyler Joynt – 4th Patient in the Stem Cell and Gene Therapy Trial on November 15, 2021 Read More »
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 …
Stem Cell Trial Phase I/II Updates by Stéphanie Cherqui, PhD Read More »
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 …
The Third Patient Was Transplanted November 16, 2020! Read More »
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