Friedreich's ataxia: the European consortium (Lancet )

Often, the development of therapeutic approaches for rare disease lags behind that of other disorders not because of poor understanding of the basic pathophysiology, but because of a paucity of systematic clinical information. The ideal way to address this scarcity of clinical knowledge and to facilitate therapeutic advancement is through controlled assessment of large numbers of patients in natural history studies across several institutions. In The Lancet Neurology, Kathrin Reetz and collegues1 report a cross-sectional analysis of baseline data for European patients with Friedreich's ataxia.