Organization: Cystinosis Research Foundation

Grants totaling more than $1.6 million were awarded by the Cystinosis Research Foundation (CRF) for 11 new research projects.

These research projects seek to cure cystinosis and advance new treatments for the rare metabolic and fatal disease. The newly funded studies include research focused on corneal cystinosis, muscle wasting and stem cells.

Cystinosis afflicts about 500 children and young adults in the United States, and about 2,000 worldwide.

The CRF is the largest provider of grants for cystinosis research in the world, funding over 103 multi-year research studies in 11 countries totaling more than $17 million. The new CRF research will be under way at universities and hospitals in the United States, France, Belgium, Switzerland and New Zealand.

CRF-funded research led to discovery of a dramatic improvement in the life-saving medication and to the first allogeneic stem cell clinical pilot study for cystinosis, which is being conducted at the Ronald Reagan UCLA Medical Center. Recently, two CRF-funded researchers were awarded NIH grants totaling more than $3 million, leveraging CRF grant money.

Discoveries made by CRF funded researchers have helped advance potential treatments for more prevalent and well-known disorders and diseases such as Huntington’s and NASH, a progressive liver disease.

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