FARA, Friedreich’s Ataxia Research Alliance, was founded by Raychel and Ron Bartek and is run by many amazing people who are devoting their time to finding a cure for FA.
FARA is a 501(c)(3) non-profit, tax exempt organization.
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The FA Treatment Pipeline is a visual tool for communicating the progress of research and development on lead therapeutic candidates.
Along the vertical axis lead candidates are grouped based on mechanism of action or approach to treatment, e.g., where or how each drug might work in the cell, technological approach, or problem being addressed. The horizontal axis indicates the stage of the research where the candidate is in development.
The first two stages, discovery and pre-clinical, take place in the research laboratory, and represent early discovery and development. The IND stage is an important milestone – this is when there is a regulatory filing with the United States Food and Drug Administration (FDA) to begin human studies. The stages “Phase 1” through “Phase 3” are phases of clinical trials/studies, when a drug is tested in individuals for safety and efficacy. With sufficient evidence proving a drug benefit, a new drug application (NDA) can be filed with the FDA. Once an NDA is approved, the drug then becomes available to individuals in their pharmacies.