FARA Mission / Organization
The Friedreich's Ataxia Research Alliance (FARA) is a national, public, 501(c)(3), non-profit, tax-exempt organization dedicated to the pursuit of scientific research leading to treatments and a cure for Friedreich's ataxia.
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.
FARA focuses on grant making for FA research and building collaborations with organizations dedicated to advancing treatments for FA. Due to the progressive nature of the disease and the promise of treatments in development, there is urgency to our efforts. Directing attention and resources to FA research and partnering with others that share this commitment, FARA believes it can help bring forward effective treatments and a cure for FA.
- Raise funds for advancing scientific research in FA.
- Facilitate a competitive and highly regarded grant making program that supports greater than 1 million dollars in research annually and focuses on translational and clinical research (moving discoveries through development to clinical improvements for patients).
- Manage an FA patient registry and support the development of a collaborative clinical network for FA clinical trials.
- Foster public-private partnerships between academic research investigators and pharmaceutical companies.
- Promote the collaborative exchange of information within the scientific community through conferences and networks.
- Work cooperatively with government entities and the other organizations that support scientific research aimed at treatments for this disorder.
- Rally patients, patient families, scientific investigators, healthcare providers, and others to be supporters and advocates for scientific advancements that will lead to treatments and a cure.
Thanks to the committed efforts of many FA scientists, we now understand the cause of FA and specific mechanisms leading to damage in patients, such as gene mutation, decreased frataxin production, iron sulfur cluster formation, and mitochondrial dysfunction. FARA is supporting the development of treatments aimed at each of these different mechanisms of damage. Because it is based on solid basic science discovery, this targeted approach to treatment has great potential. Furthermore, FA researchers believe that treatment will come in the form of a “cocktail” therapy - meaning that therapies aimed at the different mechanisms of damage have the potential to be used in conjunction with one another to treat the disorder. The graph in the right hand column depicts the different treatment approaches currently being developed with FARA's support. For further information on these research areas, visit the Research Pipeline page.
Research Ripple Effect
Dr. Harold Varmus, former Director or the National Institutes of Health, gave the following testimony before the House Commerce Subcommitte on Health and Environment in 1998:
“The story of Friedreich's ataxia illustrates how many areas of clinical and basic research can come together in unexpected ways. In this rare disorder, research involving neuroscience, genetics, clinical medicine, molecular biology, and even biology of yeast and bacteria are converging. The findings offer insights to basic biology and to many other disorders, and illustrate the importance of understanding the mechanism of disease in order to devise treatments.”
Insights into Friedreich's ataxia could prove beneficial to understandings in other diseases such as:
- Muscular Dystrophies (ALS, SMA)
- Parkinson's Disease
- Huntington's Disease
- Alzheimer's Disease
- Fragile X Syndrome
- Mitochondrial diseases (MELAS, MERF, Lebers)
- Cerebellar ataxias