Promising news re FA

[wobblybee]

🙂 Progress can be followed by logging onto clinicaltrials.gov NCT04176991

About CTI-1601

Discovered by R. Mark Payne, MD, at Indiana University School of Medicine, CTI-1601 is a recombinant fusion protein intended to deliver human frataxin into the mitochondria of patients with Friedreich’s ataxia (FA), where researchers believe it is processed to mature frataxin and becomes active in mitochondrial metabolism. Due to a genetic abnormality, patients with FA are unable to produce enough of this essential protein. CTI-1601 is the only frataxin replacement therapy in clinical development. The U.S. Food and Drug Administration (FDA) granted orphan drug status to CTI-1601, which is currently in a Phase 1 trial.

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THANK YOU WOBBLYBEE FOR STAYING ON TOP OF THE RESEARCH. YOUR INPUT IS VALUABLE TO THIS COMMUNITY.