October 24, 2006 — The Friedreich’s Ataxia Research Alliance (FARA) and the National Institutes of Health (NIH) will collaborate to fund, organize, and co-host the Third International Friedreich's Ataxia (FRDA) Scientific Research Conference at the NIH in Bethesda, Maryland, outside Washington, D.C., on November 10, 11 and 12, 2006.

Nearly 150 leading Friedreich's ataxia scientists from around the world will gather to compare research findings, share insights, and chart the course ahead in the search for treatments and a cure for FRDA.

Representatives from Friedreich's ataxia-related patient advocacy and interest groups including Ataxia UK, Friedreich's Ataxia Research Association of Australia, Muscular Dystrophy Association (MDA), National Ataxia Foundation (NAF), International Network of Ataxia Friends (Internaf), Friedreich's Ataxia Parents Group (FAPG), GoFAR (an Italian FA advocacy and research group) and EuroAtaxia will be in attendance.

We have received more than 75 abstracts from scientists requesting to present their data and findings. The focus of platform presentations will be on new or controversial data only. There will be facilitated roundtable discussions at the end of every session. The success of FARA's previous two conferences in 1999 and 2002 at the NIH has helped the scientific community focus its research on possible treatments for FRDA.

Conference attendance is closed, but at the conclusion of the conference we will share the submitted scientific abstracts via the FARA website. We will also continue to provide updates on these developments in the months ahead via the FARA website, newsletters, press releases, and other public communications.



PROGRAM SCHEDULE

Friday, November 10, 2006

Session I – The Friedreich’s Ataxia Disease Gene
Session Chair: Robert D. Wells, PhD

• GAA repeat structure
• GAA repeat instability
• Effect of GAA repeats on transcription

Session II – The Friedreich’s Ataxia Protein
Session Chair: Grazia Isaya, MD, PhD

• Biogenesis, structure, and transport
• Multimerization and iron interactions
• Mitochondrial and cellular functions
• Effects of point mutations

Session III – Iron Metabolism
Session Chair: Tracey Rouault, MD

• Iron-sulfur-cluster formation
• Iron transport
• Role(s) of iron in cell biology
• Diseases of iron metabolism

Session IV – Cellular and Mitochondrial Pathophysiology of Friedreich’s Ataxia
Session Chairs: Massimo Pandolfo, MD and Michio Hirano, MD

• Oxidative stress and antioxidant defenses
• Profiling (microarrays and proteomics)
• Non-mitochondrial iron-sulfur-cluster proteins
• Bioenergetics
• Electron-transport-chain defects
• Antioxidant defenses
• The role of mitochondria in apoptosis
• Mitochondrial diseases

Session V – Friedreich’s Ataxia Models
Session Chair: Hélène Puccio, PhD

• Lower organisms
• Mouse models
• Cell culture

Session VI – Therapeutic Approaches
Session Chair: Robert B. Wilson, MD, PhD

• Completed clinical trials
• Clinical trials in progress
• Planned clinical trials
• Drug screening and development

Session VII – Phase III Efficacy Measures
Session Chairs: Martin Delatycki, MD and David R. Lynch, MD, PhD

• Summary of biomarker meeting
• Potential surrogate endpoints
• Exercise tolerance testing
• Performance measures
• Ataxia scales