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National Ataxia Foundation

Research Pipeline

DRPLA Treatment Pipeline

Tracking progress of the development of DRPLA therapies.

Pipeline for DRPLA

This is a visual tool created to show the progress of all DRPLA therapies that are currently being developed. Along the vertical axis, treatments are grouped by their mechanism, or how the drug works. The horizontal axis shows the stage of research where the drug is in development. Phases 1 through 3 involve clinical trials with human participants. Visit our Clinical Trials page to learn more about each phase. If a clinical trial provides enough evidence to prove that the drug is safe and effective, the sponsor can file a New Drug Application (NDA) with the FDA. After an NDA is approved, the drug can become available to individuals through their healthcare provider.

Graph depicting the phase of drug development for various drugs to treat DRPLA

Please note that a drug’s status is subject to change. Go to clinicaltrials.gov for the most current information on a therapy. 

Studies that are listed as “recruiting” are looking for participants. To learn more about participating in research visit our Clinical Trials page.

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