The CRF story began in 2003 with small, dedicated group of parents who wanted to fund research to improve the lives of their children. At the time, only a handful of researchers were studying cystinosis. In 2006, we met a brilliant scientist, Dr. Stéphanie Cherqui, who believed gene therapy might one day cure cystinosis. We were in unchartered territory but we believed in Stéphanie’s vision. Our families and friends believed too and joined the quest. It was, in many ways, the perfect alignment of determination and courage: relentless parents, a committed researcher, brave patients, and a foundation willing to take bold risks in pursuit of a cure.
The publication of this paper represents a significant personal and professional milestone for Dr. Cherqui but it is also far more than academic recognition. It marks a significant scientific milestone and reflects the extraordinary persistence and excellence of Dr. Cherqui and her colleagues. For the CRF community, it is validation of more than two decades of unwavering commitment to research.
To us, Stéphanie is not only an exceptional scientist, she is a friend. Her work has given our families an immeasurable gift: hope. Hope that we can stop the progression of cystinosis. Hope that this therapy may one day be a cure.
As Novartis oversees the next phase of the trial, we eagerly await news about the first patient treated and remain steadfast in our commitment to ensuring that this therapy becomes available to every person with cystinosis.
We congratulate Dr. Cherqui and her team, and we extend heartfelt gratitude to every patient, family, and donor who made this achievement possible.
We will keep the momentum moving forward and continue to fund research that will give hope to those living with cystinosis.
