FARA's Mission is to marshal and focus the resources and relationships needed to cure FA by raising funds for research, promoting public awareness, and aligning scientists, patients, clinicians, government agencies, pharmaceutical companies and other organizations dedicated to curing FA and related diseases.
Your opportunity to tell the FDA what is important to you in finding a treatment for Friedreich’s Ataxia
Kristin Morrow was one of ten panelists that spoke at the FDA meeting to describe how the symptoms of FA effects the lives of patients and what beneficial treatments would look like.
To see Kristin, scroll to 1:32:00.
The Friedreich’s Ataxia 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.