Age at onset in genetic prion disease and the design of preventive clinical trials

OBJECTIVE: To determine whether preventive trials in genetic prion disease could be designed to follow presymptomatic mutation carriers to onset of disease. METHODS: We assembled age at onset or death data from 1,094 individuals with high penetrance mutations in the prion protein gene (PRNP) in orde...

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Detaylı Bibliyografya
Yayımlandı:Neurology
Asıl Yazarlar: Minikel, Eric Vallabh, Vallabh, Sonia M., Orseth, Margaret C., Brandel, Jean-Philippe, Haïk, Stéphane, Laplanche, Jean-Louis, Zerr, Inga, Parchi, Piero, Capellari, Sabina, Safar, Jiri, Kenny, Janna, Fong, Jamie C., Takada, Leonel T., Ponto, Claudia, Hermann, Peter, Knipper, Tobias, Stehmann, Christiane, Kitamoto, Tetsuyuki, Ae, Ryusuke, Hamaguchi, Tsuyoshi, Sanjo, Nobuo, Tsukamoto, Tadashi, Mizusawa, Hidehiro, Collins, Steven J., Chiesa, Roberto, Roiter, Ignazio, de Pedro-Cuesta, Jesús, Calero, Miguel, Geschwind, Michael D., Yamada, Masahito, Nakamura, Yosikazu, Mead, Simon
Materyal Türü: Artigo
Dil:Inglês
Baskı/Yayın Bilgisi: Lippincott Williams & Wilkins 2019
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Article
Online Erişim:https://ncbi.nlm.nih.gov/pmc/articles/PMC6656649/
https://ncbi.nlm.nih.gov/pubmed/31171647
https://ncbi.nlm.nih.govhttp://dx.doi.org/10.1212/WNL.0000000000007745
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