rare disease

How A Rare Diagnosis Impacts A Family

The Dicks Family sat down for a candid conversation with Daniel Levine from Global Genes. During the latest podcast,...

This summer we reached a milestone – over $40,000 in scholarships awarded since our program’s inception. Congratulations to the...

Please click here for the latest updates from Leadiant on the Cystadrops shortage. If you have further questions, please...

Interim CRN President, Tim Wyman, has written an open letter to the cystinosis community. You can read it here.

The goal of Rare Disease Day is to raise awareness amongst the general public and decision-makers about rare diseases and their impact on...

AVROBIO Announces 100% Kidney Substrate Reduction at 12 Months Post-Gene Therapy in...

Children’s Friend Inc. is studying the experiences in and barriers to accessing mental health services for families. For the...

November 17, 2020 – AVROBIO announced that data suggests functional and clinical improvements for the first cystinosis patient at...

Learn more about gene therapy as it relates to the cystinosis community. Dr. Paul Grimm from from Stanford University...

Let’s have an open discussion on mental health and living with cystinosis. Everyone within the community is invited! Licensed...