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Apr 4, 2014

Transgenomic to Run Genetic Tests for Raptor's Leigh Syndrome Candidate Trial

  • Transgenomic has agreed to provide genetic profiling services for Raptor Pharmaceuticals' RP103-MITO-001 trial evaluating RP103 (cysteamine bitartrate) as a possible treatment for Leigh syndrome—a severe neurological disorder caused by genetic defects in mitochondrial or nuclear DNA—and other inherited mitochondrial disorders. 

    Paul Kinnon, Transgenomic's president and CEO, said in a statement that the firm would be using its mitochondrial genetic testing capabilities to obtain the genetic profiling information Raptor needs for this trial. "This is a key example of how Transgenomic works with pharmaceutical companies to employ complementary genetic tests designed to improve clinical diagnoses and outcomes," he added.

    RP103, which is currently being marketed in the U.S. as Procysbi® delayed-release capsules for the management of nephropathic cystinosis in adults and children six years and older, is also undergoing clinical trials for Huntington's disease and nonalcoholic fatty liver disease in children. Raptor submitted an IND to the FDA for the RP103-MITO-001 trial back in December.



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