Article
Effect of Oral Valproic Acid vs Placebo for Vision Loss in Patients With Autosomal Dominant Retinitis Pigmentosa: A Randomized Phase 2 Multicenter Placebo-Controlled Clinical Trial.
JAMA ophthalmology - 1 Aug 2018
Birch David G, Bernstein Paul S, Iannacone Alessandro, Pennesi Mark E, Lam Byron L, Heckenlively John, Csaky Karl, Hartnett Mary Elizabeth, Winthrop Kevin L, Jayasundera Thiran, Hughbanks-Wheaton Dianna K, Warner Judith, Yang Paul, Fish Gary Edd, Teske Michael P, Sklaver Neal L, Erker Laura, Chegarnov Elvira, Smith Travis, Wahle Aimee, VanVeldhuisen Paul C, McCormack Jennifer, Lindblad Robert, Bramer Steven, Rose Stephen, Zilliox Patricia, Francis Peter J, Weleber Richard G
Abstract excerpt
Importance: There are no approved drug treatments for autosomal dominant retinitis pigmentosa, a relentlessly progressive cause of adult and childhood blindness. Objectives: To evaluate the potential efficacy and assess the safety of orally administered valproic acid (VPA) in the treatment of autosomal dominant retinitis pigmentosa. Design, Setting, and Participants: Multicenter, phase 2, prospective,...
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