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Rare Diseases of the Eye — Development Opportunities for Novel Therapies

Developing therapies for rare ophthalmologic disorders present a range of unique challenges — from disease characterization and determination of appropriate endpoints to trial design, patient recruitment and site selection. Advances in science, extending from genomics to real-world evidence, offer unprecedented opportunities to explore innovative research strategies that can achieve clinical trial success. This paper discusses key concerns and success factors in developing therapies for rare diseases of the eye, with a focus on PPD’s capabilities to meet the special challenges of the patient-centered rare disease research environment.

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Rare Disease