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eRAM

encyclopedia of Rare Disease Annotation for Precision Medicine




Disease leber congenital amaurosis
Comorbidity C0456909|blindness
Sentences 1
PubMedID- 25030840 Oral 9-cis retinoid for childhood blindness due to leber congenital amaurosis caused by rpe65 or lrat mutations: an open-label phase 1b trial.

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