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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

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 autoso...

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Vydáno v:JAMA Ophthalmol
Hlavní autoři: 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.
Médium: Artigo
Jazyk:Inglês
Vydáno: American Medical Association 2018
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On-line přístup:https://ncbi.nlm.nih.gov/pmc/articles/PMC6142953/
https://ncbi.nlm.nih.gov/pubmed/29879277
https://ncbi.nlm.nih.govhttp://dx.doi.org/10.1001/jamaophthalmol.2018.1171
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