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Article

Ataxia-telangiectasia clinical trial landscape and the obstacles to overcome.

Expert opinion on investigational drugs - 1 Jan 2000

Kuhn Katrina, Lederman Howard M, McGrath-Morrow Sharon A

Abstract excerpt

INTRODUCTION: Ataxia telangiectasia (A-T) is a life-limiting autosomal recessive disease characterized by cerebellar degeneration, ocular telangiectasias, and sinopulmonary disease. Since there is no cure for A-T, the standard of care is primarily supportive. AREAS COVERED: We review clinical trials available in PubMed from 1990 to 2023 focused on lessening A-T disease burden. These approaches include genetic...

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Identifiers

PMID: 37622329
DOI: 10.1080/13543784.2023.2249399
PMCID: PMC10530584

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