Challenges ahead for trials in Friedreich’s ataxia

David R Lynch, Elizabeth Kichula, The Lancet Neurology, Volume 15, Issue 13, December 2016, Pages 1300-1301, ISSN 1474-4422, doi:10.1016/S1474-4422(16)30281-2.

Nevertheless, although natural history studies such as EFACTS identify the problems in progressing with therapeutic trials in Friedreich’s ataxia, they also identify one mechanism for keeping trials small: stratification.
In all of the natural history studies of Friedreich’s ataxia including the EFACTS, individuals with longer GAA repeat lengths had faster progression.

 Challenges ahead for trials in Friedreich’s ataxia