The history of cystinosis is one of the most successful examples of rare disease research leading to meaningful treatments. In a recent interview with the European Medical Journal, William Gahl—a…
What Happened? Hansa Biopharma, a Swedish company, announced that the first patient in Australia has successfully received a kidney transplant after being treated with a new medication called Idefirix® (imlifidase).…
Health Canada has officially approved Cysklar® (cysteamine ophthalmic solution) 0.44%, marking a significant breakthrough for Canadians living with cystinosis. According to BusinessWire.com, the approval enables patients to access a topical…
Web-Based Living with Cystinosis Support Group A supportive, web-based peer support group for those living with cystinosis facilitated by Kerry Heckman, MSW, LICSW 3rd Tuesday of each month 4-5 PM…
Web-Based Living with Cystinosis Support Group A supportive, web-based peer support group for those living with cystinosis facilitated by Kerry Heckman, MSW, LICSW 3rd Tuesday of each month 4-5 PM…
Web-Based Living with Cystinosis Support Group A supportive, web-based peer support group for those living with cystinosis facilitated by Kerry Heckman, MSW, LICSW 3rd Tuesday of each month 4-5 PM…
Web-Based Living with Cystinosis Support Group A supportive, web-based peer support group for those living with cystinosis facilitated by Kerry Heckman, MSW, LICSW 3rd Tuesday of each month 4-5 PM…
Web-Based Living with Cystinosis Support Group A supportive, web-based peer support group for those living with cystinosis facilitated by Kerry Heckman, MSW, LICSW 3rd Tuesday of each month 4-5 PM…
Welcome to Study of the Week from Patient Worthy. In this segment, we select a study we posted about from the previous week that we think is of particular interest…
According to a story from SciTech Daily, a team of researchers affiliated with the University of Zurich's ITINERARE University Research Priority Program recently collaborated with Insilico Medicine to conduct research…
Note: These quotes and reflections are from members of the cystinosis community who were in attendance at the 2023 Cystinosis Research Network Family Conference which took place in Nashville, TN…
Web-Based Living with Cystinosis Support Group A supportive, web-based peer support group for those living with cystinosis facilitated by Kerry Heckman, MSW, LICSW 3rd Tuesday of each month 4-5 PM…
2023 CRN Conference July 13-15, 2023 After four years apart, we welcome you to join us in Nashville, Tennesse, lovingly known as “Music City.” In addition to being surrounded by…
Web-Based Living with Cystinosis Support Group A supportive, web-based peer support group for those living with cystinosis facilitated by Kerry Heckman, MSW, LICSW 3rd Tuesday of each month 4-5 PM…
Web-Based Living with Cystinosis Support Group A supportive, web-based peer support group for those living with cystinosis facilitated by Kerry Heckman, MSW, LICSW 3rd Tuesday of each month 4-5 PM…
Written by K.M. I have never given much thought to how I would die. Perhaps because my health and longevity has been laid out for me since infancy. Maybe I…
May 7, 2023 will be recognized as Cystinosis Awareness Day. This will be a day to spread awareness about the rare disease cystinosis among the general public and the medical…
Web-Based Living with Cystinosis Support Group A supportive, web-based peer support group for those living with cystinosis facilitated by Kerry Heckman, MSW, LICSW 3rd Tuesday of each month 4-5 PM…
According to a late October 2022 news release from gene therapy company AVROBIO, the sixth patient has been dosed in a Phase 1/2 clinical study evaluating AVR-RD-04 for patients…
In a late September 2022 news release from AVROBIO, Inc., the company’s gene therapy candidate AVR-RD-04 recently earned Rare Pediatric Disease designation. This investigational therapy is being developed as a…
For the last two and a half years, the COVID-19 pandemic has dominated the headlines. It has been hard to look online – or do anything in person – without…
Welcome to the Rare Classroom, a new series from Patient Worthy. Rare Classroom is designed for the curious reader who wants to get informed on some of the rarest, most…
This story was originally published in The Cystinosis Advocate, the newsletter for the Cystinosis Research Network, a Patient Worthy partner organization. Continued From Part Two We were told about what…
This story was originally published in The Cystinosis Advocate, the newsletter for the Cystinosis Research Network, a Patient Worthy partner organization. Continued From Part One At this stage, the ED…
This story was originally published in The Cystinosis Advocate, the newsletter for the Cystinosis Research Network, a Patient Worthy partner organization. Oscar was born in January 2020. He was a…
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