Clinical Trial Update: Safety and Efficacy Study in Subjects With Leber Congenital Amaurosis [Phase 3]

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Summary

A Phase 3 study evaluating safety and efficacy in subjects with Leber congenital amaurosis due to RPE65 mutations has posted results.

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Diseases / indications

• Inherited Retinal Dystrophy Due to Rpe65 Mutations
• Leber Congenital Amaurosis

Drugs mentioned

• Luxturna (voretigene neparvovec-rzyl)

Snippet

Sponsor: Spark Therapeutics, Inc. | Conditions: Inherited Retinal Dystrophy Due to RPE65 Mutations, Leber Congenital Amaurosis | Phase 3 | Status: ACTIVE_NOT_RECRUITING | Results posted: 2018-03-26