Pharmaceutical Company to Continue Friedreich’s Ataxia Clinical Trial

Pharmaceutical company Retrotope has announced that it’s opening enrollment for the “highest dose cohort” of an oral medication being tested on patients with Friedreich’s ataxia.

This next phase in the 28-day study follows what Retrotope called “the successful completion of the first dose cohort” of a medication called RT001. In a news announcement, Retrotope said RT001 was well tolerated, with “no serious adverse events or dose-limiting toxicities” discovered.

Retrotope described RT001 as a “chemically stabilized form of a natural fatty acid that confers resistance to lipid peroxidation in mitochondrial and cellular membranes via a novel mechanism.”

The study is being carried out at the University of South Florida Ataxia Research Center in Tampa, and at the Collaborative Neuroscience Network in Long Beach, Calif.

The Friedreich’s Ataxia Research Alliance (FARA) calls the condition “a debilitating, life-shortening, degenerative neuromuscular disorder” most often diagnosed in children and teens. There are currently no treatments for Friedreich’s ataxia.

Jennifer Farmer, MS, CGC, the executive director of the Friedreich's Ataxia Research Alliance, said in the news announcement, "FARA is pleased to continue our support of Retrotope and this clinical study of a new approach to treat Friedreich’s ataxia. We are excited by the potential of RT001, and we are grateful to the individuals who have volunteered for this study."

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Laurie Watanabe is the editor of Mobility Management. She can be reached at

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