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Safety, tolerability, and pharmacokinetics of antisense oligonucleotide BIIB078 in adults with C9orf72-associated amyotrophic lateral sclerosis: a phase 1, randomised, double blinded, placebo-controlled, multiple ascending dose study

Leonard H. van den Berg, Jeffrey D. Rothstein, Pamela J. Shaw, Suma Babu, Michael Benatar, Robert C. Bucelli, Angela Genge, Jonathan D. Glass, Orla Hardiman, Vincenzo Libri,

The Lancet Neurology · 2024 · 70 citations · 11 references

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11

A Hexanucleotide Repeat Expansion in C9ORF72 Is the Cause of Chromosome 9p21-Linked ALS-FTD

Alan E. Renton, Elisa Majounie, Adrian J. Waite et al. · Neuron · 2011 · 4.5K citations · Full text

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Frequency of the C9orf72 hexanucleotide repeat expansion in patients with amyotrophic lateral sclerosis and frontotemporal dementia: a cross-sectional study

Elisa Majounie, Alan E. Renton, Kin Y. Mok et al. · The Lancet Neurology · 2012 · 1.2K citations · Full text

, , +15

Comparison of three analytical platforms for quantification of the neurofilament light chain in blood samples: ELISA, electrochemiluminescence immunoassay and Simoa

Jens Kühle, Christian Barro, Ulf Andréasson et al. · Clinical Chemistry and Laboratory Medicine (CCLM) · 2016 · 739 citations

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Gain of Toxicity from ALS/FTD-Linked Repeat Expansions in C9ORF72 Is Alleviated by Antisense Oligonucleotides Targeting GGGGCC-Containing RNAs

Jie Jiang, Qiang Zhu, Tania F. Gendron et al. · Neuron · 2016 · 540 citations · Full text

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Neurofilament levels as biomarkers in asymptomatic and symptomatic familial amyotrophic lateral sclerosis

Patrick Weydt, Patrick Oeckl, André Huss et al. · Annals of Neurology · 2015 · 221 citations

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