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CLINICAL_TRIALS trial_result · 2026-06-04

Clinical Trial Update: Study of Efficacy and Safety of Voretigene Neparvovec in Japanese Patients With Biallelic RPE65 Mutation-associated Retinal Dystrophy [Phase 3]

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Summary

A Phase 3 trial evaluating the efficacy and safety of voretigene neparvovec in Japanese patients with biallelic RPE65 mutation-associated retinal dystrophy has …

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Diseases / indications

Drugs mentioned

  • Voretigene Neparvovec (voretigene neparvovec)

Snippet

Sponsor: Novartis Pharmaceuticals | Conditions: Biallelic RPE65 Mutation-associated Retinal Dystrophy | Phase 3 | Status: COMPLETED