Cystinosis Research Network

10/15/2026

- Rare Disease News -

The FDA has launched an initiative to develop a new regulatory framework designed around the unique realities of rare disease drug development. This includes those with limited natural history data and challenges with traditional clinical trial designs.

The goal is not to lower standards for safety or effectiveness, but to ensure the methods used to evaluate potential treatments are appropriate for rare diseases.

If successful, this could lead to more consistent and effective ways to develop new therapies treatment options for people living with rare diseases like .

Read more from National Organization for Rare Disorders, Inc. (NORD): https://tinyurl.com/2p54anaw

Statement from Pam Gavin, President and Chief Executive Officer

10/13/2026

“When you have a child with a chronic condition, you're of course fearful and scared about what the future will hold for them. We had to come to the realization that life isn’t going to go exactly as we had planned. For those who are wading through the uncertainty of a diagnosis, I want to extend the biggest hugs. Slowly, but surely, you do figure it out, and it does get better.”
- Erin, mom to Sofie

https://tinyurl.com/43vh673n

A life-changing rare disease diagnosis and complex treatment bring Sofie and her family to CHOP for multidisciplinary care. As a baby, Sofie was diagnosed with a rare condition called cystinosis. “When I think of Sofie, the first thing I think of is joy,” said Erin. Erin is the mother of a vibra...

10/12/2026

Who on your care team should know about this?

Research and advances in cystinosis care are continually evolving. The CRN Scientific Symposium brings healthcare professionals, researchers, and scientists together to share knowledge and learn from colleagues.

Is there an agenda topic you’d like your care team to hear more about? Connecting them with this event could help broaden the knowledge informing your care and support conversations about your needs.

Share the symposium with them today! Details here: https://cystinosis.org/event/2026symposium/

CRN Scientific Symposium
Advancing the Future of Cystinosis Care
November 13, 2026 Ann Arbor, MI

10/08/2026

Agree or disagree?

If it's out of your hands, it should be free from your mind, too.

10/08/2026

Where are our cystinosis warriors searching for a remote, part-time job?

RARE Foundation (formerly EveryLife Foundation for Rare Diseases) is looking for a Communications Assistant. You could be their next intern!

Info at https://tinyurl.com/bd63479h

The RARE Foundation Is Hiring a Communications Assistant!

Join our dedicated team in making a meaningful difference in the lives of more than 30 million Americans living with rare diseases.

At the RARE Foundation, we are committed to improving the lives of people and families affected by more than 10,000 known rare diseases through science, policy, and advocacy.

We’re currently looking for a Communications Assistant to join our team and support our communications efforts. If you or someone you know may be interested, please share this opportunity or visit our website to apply! https://hubs.li/Q04zyXcs0

10/06/2026

See you at 8pm eastern!
(Zoom link in comments)

10/05/2026

Tues, October 6
8p et / 7p ct / 5p pt
Cystinosis and Aging

Join co-hosts Cheryl and Hannah for a live discussion. Zoom link is in the comments.

We hope to see you there!

09/29/2026

“Rare doesn’t mean less important. It means not enough people know about it.” -Gail Devers

Global Genes

09/28/2026

Rounding out our 2026 awardees is Miss Jordan.
This sibling scholarship recipient is pursuing a Masters in Forensic and Investigative Sciences.

Way to go, Jordan!