Methodological insights for randomized clinical trials of retinitis pigmentosa: lessons learned from a trial of valproic acid

BP Brooks, B Jeffrey - JAMA ophthalmology, 2018 - jamanetwork.com
Retinitis pigmentosa (RP) is a genetically heterogeneous group of photoreceptor
degenerations for which no treatments exist. In this issue of JAMA Ophthalmology, Birch and
colleagues1 report on the repurposing of oral valproic acid as a potential treatment for
autosomal dominant RP. The authors found that valproic acid made the progression of
isopter III4e visual field deteriorate faster than those in the placebo group. Although at first
glance, one might think that there is little to say in commenting on a study that did not have a …