Cystinosis News and Research

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Cystinosis is a condition in which the body accumulates the amino acid cystine (a building block of proteins) within cells. Excess cystine forms crystals that can build up and damage cells. These crystals negatively affect many systems in the body, especially the kidneys and eyes.
Existing drug for rare kidney disease could benefit patients with mitochondrial disorders

Existing drug for rare kidney disease could benefit patients with mitochondrial disorders

Single infusion of wildtype HSPCs halts cellular damage caused by neuromuscular disorder in mice

Single infusion of wildtype HSPCs halts cellular damage caused by neuromuscular disorder in mice

CIRM approves $5.2 million for research on life-long treatment for rare childhood disease

CIRM approves $5.2 million for research on life-long treatment for rare childhood disease

R. Bryan Miller Symposium to spotlight special focus on rare disease research

R. Bryan Miller Symposium to spotlight special focus on rare disease research

Scientists identify new cellular pathway affected in cystinosis

Scientists identify new cellular pathway affected in cystinosis

Cystinosis Research Foundation granted $2.15M in 2013 to researchers investigating cure for cystinosis

Cystinosis Research Foundation granted $2.15M in 2013 to researchers investigating cure for cystinosis

Tackling orphan diseases: an interview with Damian Marron, CEO, TxCell

Tackling orphan diseases: an interview with Damian Marron, CEO, TxCell

Sigma-Tau Pharmaceuticals recognized as a rare disease pioneer

Sigma-Tau Pharmaceuticals recognized as a rare disease pioneer

University of Sunderland pharmaceutical scientists receive CFUK grant for research into cystinosis

University of Sunderland pharmaceutical scientists receive CFUK grant for research into cystinosis

Researchers characterize novel lysosomal PQ protein linked to cystinosis

Researchers characterize novel lysosomal PQ protein linked to cystinosis

Robert Gordon University scientists to develop new treatment for cystinosis

Robert Gordon University scientists to develop new treatment for cystinosis

Raptor reopens enrollment in DR Cysteamine Phase 3 trial to treat patients with nephropathic cystinosis

Raptor reopens enrollment in DR Cysteamine Phase 3 trial to treat patients with nephropathic cystinosis

Raptor completes enrollment in DR Cysteamine Phase 3 clinical trial in cystinosis

Raptor completes enrollment in DR Cysteamine Phase 3 clinical trial in cystinosis

New 'magic bullet' can eradicate side effects of drug used to treat cystinosis

New 'magic bullet' can eradicate side effects of drug used to treat cystinosis

Raptor initiates dosing in DR Cysteamine Phase 2 clinical trial for Huntington's Disease

Raptor initiates dosing in DR Cysteamine Phase 2 clinical trial for Huntington's Disease

Raptor Pharmaceutical updates on cysteamine bitartrate Phase 3 clinical trial in cystinosis patients

Raptor Pharmaceutical updates on cysteamine bitartrate Phase 3 clinical trial in cystinosis patients

Raptor Pharmaceutical to raise $15 million through PIPE financing

Raptor Pharmaceutical to raise $15 million through PIPE financing

Raptor partners with Uni Pharma to commercialize Convivia in Taiwan

Raptor partners with Uni Pharma to commercialize Convivia in Taiwan

Raptor initiates enrollment in pivotal Phase 3 clinical trial of DR Cysteamine for nephropathic cystinosis

Raptor initiates enrollment in pivotal Phase 3 clinical trial of DR Cysteamine for nephropathic cystinosis

EMA COMP grants orphan medicinal product designation for Raptor's DR Cysteamine for cystinosis

EMA COMP grants orphan medicinal product designation for Raptor's DR Cysteamine for cystinosis