cystinosisrn

A Cystinosis Journey by Gail Potts

My daughter, Deanna Lynn, was diagnosed with cystinosis 46 years ago in Houston, TX. At that time I did...

The great givers in my life are my family. I was born with a rare kidney disease known within...

What started as a cystinosis awareness effort turns into a life-changing encounter. It all started with Jana (my twin...

For the fifth consecutive year, the CRN is a proud participant in Giving Tuesday – a day encouraging people...

Global Genes 2019 RARE Patient Advocacy Summit was attended by members of our cystinosis community. Natasha Faisca and Clinton...

AVROBIO has released a brochure providing detail on investigational gene therapies for lysosomal storage disorders. Earlier this year, AVROBIO’s...

AVROBIO has announced the first patient has been dosed in the company’s Phase 1/2 trial of AVR-RD-04, an investigational...

This three part series is a one-of-a-kind resource for children AND adults curious about cystinosis. Each installment was carefully...

People from around the world joined the CRN Family Conference this summer to help “Ring in the Future”. Here...

NoMuRiC (Nonsense Mutation Readthrough in Cystinosis) A Phase II Clinical Trial of ELX-02 for Treatment of Cystinosis (McGill University...