FARA takes a unique approach to helping pharmaceutical companies research and develop cures for rare diseases. We have partnered with more than 10 companies interested in treating FA. As a rare disease we understand concern about patient recruitment for trials and as a neurodegenerative disease concern about validated outcome measures. Consequently, FARA has worked to develop a comprehensive clinical infrastructure to support trials and research.
FARA is a worldwide leader in growing and supporting the FA research community through our robust research grant program, international conferences and advocacy and partnership with government organizations and pharmaceutical companies. We have invested more than $15 million, through a competitive grant program, and leveraged about the same amount from government agencies, other non-profit organizations and academic partners to advance the basic and discovery science, which has led to:
- advanced understanding of genetic etiology of FA
- cellular dysfunction and pathways impacted in FA
- discovery of animal and cellular models
- validated clinical outcome measures and biomarkers
Working with FARA to develop drugs for FA and related conditions makes a lot of sense because of what we bring to the partnership. FARA is committed to helping cut preclinical and clinical development time to a minimum and getting products to market faster and at a lower cost. For more information on the resources FARA provides, please follow the links below:
Robust Research Pipeline
This pipeline illustrates both academic and industry efforts in drug discovery and development.
Drug Development Tools
FARA has funded discovery scientists working on animal and cellular models, development and validation
of high-throughput assays and worked with physicians to facilitate the creation of biorepositories.
Global Patient Registry
FARA created and maintains the largest and only worldwide registry of FA patients that currently has more than 1400 registered patients.
FARA has successfully recruited patients for 7 trials,representing all three phases of clinical trials, within only a few weeks
rather than the months or years usually required in other diseases. One phase three study recruited more patients than the
target enrollment (50 subjects) in two weeks of registry announcement which led the company to expand enrollment to 70 patients,
increasing statistical power for the study. FARA then recruited the additional 20 patients in two weeks.
Clinical Infrastructure
The Collaborative Clinical Research Network in Friedreich's Ataxia (CCRN in FA) is a growing international network,
currently of a dozen clinical research centers that work together to advance treatments and clinical care for individuals with FA.
The network collaborates with pharmaceutical companies, government agencies, other research centers and the patient community
to facilitate clinical research and trials needed to identify new therapies. The CCRN in FA has also collected natural history data
on a large cohort of FA patients, developed and validated the Friedreich’s Ataxia Rating Scales (FARS) and the functional measures
being used in FA clinical trials and is developing a variety of FA biomarkers.
Scientific Publications
FARA captures the most recent publications and scientific news related to FA.
4th International Friedreich’s Ataxia Scientific Conference
May 5th-7th 2011,
Strasbourg France
Worldwide Network of Clinicians and Researchers
The sun never sets on FARA-funded researchers and clinicians. The FARA research network spans the globe.
FARA supports clinical centers across the United States, Canada and Australia and collaborates fully with such centers throughout Europe.
These centers include physicians and researchers who know FA well, see FA patients regularly and are conducting FA clinical trials.
These centers are collaborating fully with pharmaceutical industry partners and are eager to partner with additional companies
to advance additional therapeutic candidates.
Another service FARA has effectively provided our pharmaceutical partners is making necessary recommendations and introductions to
researchers and clinicians to advance your program objectives. We can facilitate initial introductions through teleconferences or face-to-face meetings.
Thank you for your interest in FARA and Friedreich’s ataxia. We would like to meet with you personally to explore how we can
work together to bring to market promptly products that treat FA and show tremendous promise in treating many other diseases as well.
Working together, we can cure this terrible disease, through advancing to market products in FARA’s pipeline or products in your
development pipeline, faster than thought possible.
Contact FARA: Jennifer Farmer, Executive Director
jen.farmer@curefa.org
(484) 879-6160
CEO of the Pharmaceutical Research and Manufacturers of America (PhRMA), Billy Tauzin paid touching tribute to FARA
and appealed to his pharmaceutical colleagues to assist our efforts to treat and cure this rare disease.
FARA has been recognized time and again as an excellent industry partner.
Based on our track record working with government agencies and pharmaceutical companies like yours,
the NIH has cited FARA as a model organization for conducting collaborative research and forming business partnerships.
And, in fact, a recent report by the Institute of Medicine presented FARA as an ideal partner
for helping accelerate research and development in rare diseases.
“Pharmaceutical Executive Magazine” featured FARA in its
May 2010 article
on rare diseases.
info@cureFA.org|www.cureFA.org|(484) 879-6160