Treatments in Phase 1 Trials for FA


CTI-1601 is an investigational therapy designed to deliver the frataxin protein to the mitochondria of Friedreich’s ataxia patients. Dosing was completed in a Phase 1 trial in December 2020, and top-line data are expected in the second quarter of 2021.

Read more on CTI-1601


Methylprednisolone is a steroid used to reduce inflammation, and may be able to help ease the symptoms of Friedreich’s ataxia. A Phase 1 trial was completed in 2019, but it did not meet its primary objective, with results showing no clear benefit for FA patients. No information is available yet on further studies.

Read more on Methylprednisolone
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Visit the Friedreich’s Ataxia News forums to connect with others in the FA community to discuss issues, ask questions, and share your experiences related to scientific research and clinical trials.

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