Challenges associated with clinical trials for inherited and orphan retinal diseases.
Fishman
Gerald A
GA
Department of Ophthalmology and Electrophysiology at the University of Illinois, Chicago Eye Center, Chicago, IL, USA.
D002986:Clinical Trials as Topic; D015785:Eye Diseases, Hereditary / Q000235:genetics / Q000628:therapy*; D006801:Humans; D018579:Patient Selection; D035583:Rare Diseases / Q000235:genetics / Q000628:therapy*; D012164:Retinal Diseases / Q000235:genetics / Q000628:therapy*; D016896:Treatment Outcome