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Exploring microperimetry and autofluorescence endpoints for monitoring disease progression in PRPF31-associated retinopathy.

Danial RoshandelJennifer A ThompsonJason CharngDan ZhangEnid ChelvaSukanya ArunachalamMary S AttiaTina M LameyTerri L McLarenJohn N De RoachDavid A MackeySteve D WiltonSue FletcherSamuel McLenachanFred K Chen
Published in: Ophthalmic genetics (2020)
Our findings suggest that in the studied cohort, the optimal window for therapeutic intervention is the second decade of life and residual EZ span and HAR area can be considered as efficacy outcome measures. Further studies on larger samples with different PRPF31 mutations and longer follow-up duration are recommended.
Keyphrases
  • randomized controlled trial
  • case control