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Annals of Clinical and Translational Neurology|January 11, 2022
A Phase 1 study of GDC-0134, a dual leucine zipper kinase inhibitor, in ALSJonathan S Katz, Jeffrey D Rothstein, Merit E Cudkowicz, et al.BMC Neurology|December 15, 2006
Analysis of IFT74 as a candidate gene for chromosome 9p-linked ALS-FTDParastoo Momeni, Jennifer Schymick, Shushant Jain, et al.Neuron|November 1, 2024
Inhibition of RNA splicing triggers CHMP7 nuclear entry, impacting TDP-43 function and leading to the onset of ALS cellular phenotypesNorah Al-Azzam, Jenny H To, Vaishali Gautam, et al.Science Translational Medicine|October 27, 2021
Ribonuclease recruitment using a small molecule reduced c9ALS/FTD r(G<sub>4</sub>C<sub>2</sub>) repeat expansion in vitro and in vivo ALS modelsJessica A Bush, Haruo Aikawa, Rita Fuerst, et al.Plos One|May 21, 2014
Safety, pharmacokinetic, and functional effects of the nogo-a monoclonal antibody in amyotrophic lateral sclerosis: a randomized, first-in-human clinical trialVincent Meininger, Pierre-François Pradat, Andrea Corse, et al.The Lancet. Neurology|July 26, 2024
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 studyLeonard H van den Berg, Jeffrey D Rothstein, Pamela J Shaw, et al.Science Translational Medicine|September 14, 2022
Poly(ADP-ribose) promotes toxicity of <i>C9ORF72</i> arginine-rich dipeptide repeat proteinsJunli Gao, Quinlan T Mewborne, Amandeep Girdhar, et al.Neuron|August 19, 2014
Discovery of a biomarker and lead small molecules to target r(GGGGCC)-associated defects in c9FTD/ALSZhaoming Su, Yongjie Zhang, Tania F Gendron, et al.Medrxiv : the Preprint Server for Health Sciences|February 6, 2026
Is <i>SORL1</i> a common genetic target across neurodegenerative diseases?: A multi-ancestry biobank scale assessmentMarzieh Khani, Sheila N Yeboah, Catalina Cerquera-Cleves, et al.Brain : a Journal of Neurology|May 26, 2026
Is SORL1 a common genetic target across neurodegenerative diseases? A multi-ancestry biobank studyMarzieh Khani, Sheila N Yeboah, Catalina Cerquera-Cleves, et al.Pageof 21