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 successful primary outcome; however, we submit that there is much to be learned from this article.
Brooks BP, Jeffrey B. Methodological Insights for Randomized Clinical Trials of Retinitis Pigmentosa: Lessons Learned From a Trial of Valproic Acid. JAMA Ophthalmol. 2018;136(8):857–858. doi:10.1001/jamaophthalmol.2018.1167
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