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-Reference-Cited by-同舟云学术

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

Published:2024-09 Issue:9 Volume:23 Page:901-912
ISSN:1474-4422
Container-title:The Lancet Neurology
language:en
Short-container-title:The Lancet Neurology

Author:

van den Berg Leonard H,Rothstein Jeffrey D,Shaw Pamela J,Babu Suma,Benatar Michael,Bucelli Robert C,Genge Angela,Glass Jonathan D,Hardiman Orla,Libri Vincenzo,Mobach Theodore,Oskarsson Björn,Pattee Gary L,Ravits John,Shaw Christopher E,Weber Markus,Zinman Lorne,Jafar-nejad Paymaan,Rigo Frank,Lin Luan,Ferguson Toby A,Gotter Anthony L,Graham Danielle,Monine Michael,Inra Jennifer,Sinks Susie,Eraly Satish,Garafalo Steve,Fradette Stephanie

Funder

Biogen Inc

Publisher

Elsevier BV

Reference21 articles.

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3. Frequency of the C9orf72 hexanucleotide repeat expansion in patients with amyotrophic lateral sclerosis and frontotemporal dementia: a cross-sectional study;Majounie;Lancet Neurol,2012

4. A hexanucleotide repeat expansion in C9ORF72 is the cause of chromosome 9p21-linked ALS-FTD;Renton;Neuron,2011

5. Genetic epidemiology of amyotrophic lateral sclerosis: a systematic review and meta-analysis;Zou;J Neurol Neurosurg Psychiatry,2017

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