Significant improvement observed in X-linked retinitis pigmentosa treated with AAV-RPGR

Nine-month data from a phase 1/2 clinical trial of AAV-RPGR gene therapy for X-linked retinitis pigmentosa showed clinically meaningful improvement in retinal sensitivity and vision-guided mobility.
“The most significant finding is that treatment can result in an improvement in vision in a way that went beyond our expectations,” Michel Michaelides, MD, said at the virtual Euretina congress.
Disease-causing variants in the RPGR gene account for more than 70% of cases of X-linked retinitis pigmentosa and up to 20% of all cases of retinitis pigmentosa. AAV-RPGR (MeiraGTx/Janssen

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