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The risks of converting post-hoc findings into primary outcomes in subsequent trials

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Autores

Rodrigues, Filipe Brogueira

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Resumo(s)

Huntington’s disease (HD) is a devastating neurodegenerative condition caused by a triplet repeat expansion of the Huntingtin gene. Although rare it is amongst the most frequent autosomal dominant causes of dementia, frequently affecting individuals in the most productive decades of their lives. Clinically, it is characterized by a classic triad of fluctuating neuropsychiatric symptoms, and progressive movement and cognitive disorders, accompanied by other symptoms such as weight loss and sleep impairment. It is severely debilitating, has a huge impact on quality of life and is fatal, with a median survival after motor onset of around a quarter of a century.

Descrição

© Annals of Translational Medicine. All rights reserved. This journal is a peer reviewed, open access journal. All content of the journal is published under the Creative Commons Attribution-NonCommercial-NoDerivatives 4.0 International License (CC BY-NC-ND 4.0).

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Contexto Educativo

Citação

Ann Transl Med. 2019 Dec;7(Suppl 8):S337

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