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US FDA Grants Orphan Drug Designation for Retrotope’s RT001 in the treatment of Friedreich’s ataxia

First-in-human clinical trial for RT001 is now fully enrolled

LOS ALTOS, CA, June 1, 2016 – Retrotope announced today that the U.S. Food and Drug Administration (FDA) Office of Orphan Products Development granted orphan drug designation for its stabilized fatty acid drug (RT001) for the treatment of Friedreich’s ataxia (FA). This follows the recent announcement that RT001 was well tolerated with no serious adverse events or dose limiting toxicities in the first cohort of its Phase 1/2 clinical trial in FA patients.

Read the entire Press Release HERE




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