The foundation has awarded five grants worth $776,693 to the cystinosis researchers globally in its second round of proposals in 2011.
Cystinosis is a lysosomal storage disease characterized by the abnormal accumulation of the amino acid cystine. It is a genetic disorder that typically follows an autosomal recessive inheritance pattern.
The funding included additional research on stem cell and gene therapy, which holds the greatest promise to cure the rare, deadly metabolic and genetic disease.
The CRF’s autumn grants were awarded to five research programs at hospitals and universities in the US, Italy, Belgium, Ireland and Australia.
Raptor Pharmaceuticals, which has licensed the CRF-funded research, is conducting the clinical trials and anticipates FDA approval in 2012. In addition, the cysteamine and its delayed-release formula is being used in clinical trials for Huntington’s disease and NASH, a progressive liver disease.
Nancy Stack, CRF Trustee and president, said: “Acure is within sight. These studies will provide important data to realize our goals which will be life changing for the cystinosis community. Our hope is to begin transplanting healthy stem cells into human patients soon. The CRF is funding other important cystinosis research that could offer hope to those diagnosed with Parkinson's and Huntington's disease.
“We are guided by the extraordinary members of the CRF Scientific Review Board who evaluate and analyze every research proposal we receive during our twice-a-year announcement for research and fellowship applications.”
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