Cystinosis Research Foundation Facebook ad: “Hematopoietic Stem-Cell Gene Therapy for Cystinosis | NEJM”

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In our small but powerful global community of 2,500 patients, we have moved mountains together. We have paved a path to better treatments and a cure for cystinosis. The CRF cystinosis story is truly remarkable; it began in 2003 with a small, dedicated group of parents who wanted to fund research to improve the lives of their children. At that time, there were only a handful of researchers in the world studying cystinosis but we had HOPE. In 2006, we were introduced to Stephanie Cherqui, a brilliant and compassionate researcher who believed she might be able to cure cystinosis. We were in unchartered territory but we believed in Stephanie. Our families and friends believed too and joined the quest. It felt like the “perfect storm” - relentless parents, a deeply committed researcher and courageous patients who were willing to volunteer for the treatment and a community that refused to give up. Today, we have incredible news to share – Stephanie Cherqui’s groundbreaking paper, “Hematopoietic Stem-Cell Gene Therapy for Cystinosis” has been published in the New England Journal of Medicine (NEJM). The NEJM is the most prestigious, influential, and respected medical journals in the world. Publication in the journal is considered the “hallmark of academic success in medicine.” The NEJM receives over 16,000 submissions annually, with an acceptance rate of only approximately 5%. The journal publishes high-impact clinical research and is read by more than a million people globally. Just imagine – our community’s story, is now being shared around the world! This publication represents a significant personal and professional milestone for Stephanie and reflects the exceptional quality and excellence of Stephanie’s work as a researcher. We know Stephanie as a researcher but she is so much more to this community. She is also a friend. She has impacted each one of us personally, and given us hope that we will be able to stop the progression of cystinosis and that this treatment might be a cure. Congratulations to Stephanie and her colleagues who made this achievement possible. Thank you to every family, patient and donor who has been part of the CRF story. As Novartis oversees the next phase of the trial, we eagerly await news about the first patient treated. Our commitment remains unwavering – we will continue working tirelessly to ensure that this treatment becomes available to every patient living with cystinosis. Together we will keep moving forward! https://www.nejm.org/doi/full/10.1056/NEJMoa2506431

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Hematopoietic Stem-Cell Gene Therapy for Cystinosis | NEJM

Cystinosis is a multisystemic lysosomal storage disorder caused by pathogenic variants in CTNS, the gene encoding cystinosin, a lysosomal transmembrane cystine transporter. In patients with cystino...