Treatment Pipeline

This information is publicly available and reshared here for informational purposes only. It is not medical advice. FARA does not endorse, control, or govern the research pipeline programs.

Potential treatments for FA are grouped based on mechanism of action and stage of development (in the country where the candidate is furthest in its development process).

FARA helps advance treatments through funding, advocacy, patient engagement and/or collaboration. We believe the most effective treatment will likely be a combination of two or more therapies. To learn more about the stages of drug development, click here.

Active – programs currently under development and supported by the listed sponsor.
Inactive – programs no longer in development because of lack of safety or efficacy or for which the sponsor has paused recruitment because of resource constraints and for which there is not a clear path forward.