[Hereditary retinal pathologies and AMD: new therapeutic perspectives]

Area of Science:

• Ophthalmology
• Genetics
• Cell Biology

Context:

• Hereditary retinal degeneration and wet age-related macular degeneration (AMD) are significant causes of vision loss.
• Current understanding of hereditary retinal degeneration pathophysiology and promising animal model therapeutics necessitate refined clinical trial preparation.
• Advancements in anti-angiogenic therapies are progressing for neovascular AMD.

Purpose:

• To outline the essential prerequisites for successful clinical trials in hereditary retinal degeneration and wet AMD.
• To emphasize the need for standardized longitudinal follow-up and international data sharing.
• To highlight the importance of phenotyping standardization and center certification for multicenter studies.

Summary:

• Standardized longitudinal follow-up using electrophysiological, psychophysical, and morphological analyses is crucial for patient selection in hereditary retinal degeneration trials.
• International databases and standardized procedures are required to evaluate examination relevance and identify limitations for prospective studies.
• Clinical trials for neovascular AMD are advancing, though the transition to atrophic forms remains unclear.

Impact:

• Establishes a framework for preparing patients with retinal diseases for future clinical trials.
• Promotes the development of international standards for data collection and analysis in ophthalmology research.
• Facilitates the advancement of therapeutic strategies for debilitating retinal conditions like AMD and hereditary degenerations.