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Article

High-content drug screening for rare diseases.

Journal of inherited metabolic disease - 1 Jul 2017

Bellomo F, Medina D L, De Leo E, Panarella A, Emma F

Abstract excerpt

Per definition, rare diseases affect only a small number of subjects within a given population. Taken together however, they represent a considerable medical burden, which remains poorly addressed in terms of treatment. Compared to other diseases, obstacles to the development of therapies for rare diseases include less extensive physiopathology knowledge, limited number of patients to test treatments, and poor...

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Identifiers

PMID: 28593466
DOI: 10.1007/s10545-017-0055-1

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PubMed metadata and Literature Corpus relationships. Corpus snapshot updated 7 Sept 2026. Manifest publication-graph-cohort-2026-09-07.1.

Source receipt: pubmed, retrieved 7 Sept 2026.

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