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

Ngolela E. Babady, Nadege Carelle, Robert D. Wells, Tracey A. Rouault, Michio Hirano, David R. Lynch, Martin B. Delatycki, Robert B. Wilson, Grazia Isaya, Hélène Puccio

Research output: Contribution to journalReview articlepeer-review

52 Scopus citations

Abstract

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.

Original languageEnglish (US)
Pages (from-to)23-35
Number of pages13
JournalMolecular genetics and metabolism
Volume92
Issue number1-2
DOIs
StatePublished - Sep 2007

Keywords

  • Drug therapy
  • Frataxin
  • Friedreich's Ataxia
  • Iron metabolism
  • Mitochondria

ASJC Scopus subject areas

  • Endocrinology, Diabetes and Metabolism
  • Biochemistry
  • Molecular Biology
  • Genetics
  • Endocrinology

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