There are no approved drug treatments for autosomal-dominant retinitis pigmentosa.
A study of oral Valproic acid (VPA) does not support the use of VPA in the treatment of autosomal-dominant retinitis pigmentosa.
The lessons learned from a trial of Valproic acid are:
- Rigorous evaluation of drug treatment for retinitis pigmentosa is necessary.
- FDA-approved drugs for other uses can have major adverse effects.
- Patients vary greatly in their rates of progression of retinitis pigmentosa with identical gene mutations.
“Methodological Insights for Randomized Clinical Trials of Retinitis Pigmentosa”
Lessons Learned from a Trial of Valproic Acid
JAMA Ophthalmol 136:857-858, August 2018
Brian P. Brooks, MD, PhD; Brett Jeffrey, PhD