Concept paper on the need for the guideline on the clinical investigation of medicinal products for the treatment of inherited retinal dystrophies - Scientific guideline

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Keywords: Inherited retinal dystrophies (IRD), Retinitis Pigmentosa (RP), Leber Congenital Amaurosis (LCA), clinical development, gene therapy, gene-agnostic (broad) therapy

Open consultation

Concept paper on the need for the guideline on the clinical investigation of medicinal products for the treatment of inherited retinal dystrophies

Draft: consultation open Consultation dates: to Reference Number: EMA/219696/2026 Summary:

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English (EN) (198.01 KB - PDF)

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