Welcome to theCystinosis Research Network

Cystinosis is a rare, genetic, metabolic, lysosomal storage disease that causes an abnormal accumulation of the amino acid cystine in various organs and tissues of the body such as the kidneys, eyes, muscles, pancreas and brain.

The cystine accumulation causes widespread tissue and organ damage. Cystine accumulation can lead to kidney failure, muscle wasting, swallowing difficulty, diabetes, hypothyroidism, cerebral atrophy, photophobia, blindness, corneal ulceration, ventilatory impairment, and more. Without treatment, children with cystinosis will usually develop end stage kidney failure and die prematurely.

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Cystinosis Warrior Impact Program 

No matter where you are within the cystinosis journey, we can match you with resources to help. Let’s break down those barriers together.

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Health Care Professionals

The CRN provides the latest updates on research and treatment advancements, funds research grants and provides best clinical practices resources.

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AVROBIO Reports Positive Data From Phase 1/2 Clinical Trial...

Press release originally published May 17, 2022 on Avrobio.com. New early data show...

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Cystinosis Warrior Impact...

In honor of 5th Annual Cystinosis Awareness Day a new global initiative has been announced: the Cystinosis Warrior Impact Program.  ...

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Newly Diagnosed

You’ve received a diagnosis. Now what?

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Daily life with cystinosis can present challenges. There are resources to help you along the way.

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Research Developments

Teams of medical experts are researching every aspect of cystinosis with the purpose of understanding our disease, finding improved treatments and a cure.

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