Advancements in the pathophysiology of Friedreich's Ataxia and new prospects for treatments

Mol Genet Metab. 2007 Sep-Oct;92(1-2):23-35. doi: 10.1016/j.ymgme.2007.05.009. Epub 2007 Jun 26.


On November 9-12, 2006, the Friedreich's Ataxia Research Alliance (FARA) and the National Institutes of Health (NIH) hosted the Third International Friedreich's Ataxia (FRDA) Scientific Conference at the NIH in Bethesda, Maryland, highlighting the exciting research leading now to a variety of clinical trials that show promise of effective treatments for this devastating disorder. Nearly 150 leading FRDA scientists from around the world discussed their new insights and findings. The presence of six pharmaceutical and biotechnology companies underscored the importance of the public-private partnership that has grown in the past years. Some of these companies are already involved in advancing promising drug compounds into clinical trials, while others are eager to help take newer discoveries through drug development and into subsequent clinical trials. National Institute of Neurological Disorders and Stroke (NINDS) Director Dr. Story Landis noted in her opening remarks for the conference that there was a "palpable sense of energy, excitement, and enthusiasm" over the scientific progress made since the FRDA gene was discovered over 10 years ago.

Publication types

  • Research Support, N.I.H., Extramural
  • Research Support, Non-U.S. Gov't
  • Review

MeSH terms

  • Clinical Trials as Topic
  • Drug Industry
  • Friedreich Ataxia / physiopathology*
  • Friedreich Ataxia / therapy*
  • Humans
  • National Institutes of Health (U.S.)
  • Trinucleotide Repeats / genetics
  • United States