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Annals of Clinical and Translational Neurology|April 25, 2015
Identification of distinct circulating exosomes in Parkinson's diseasePaul R Tomlinson, Ying Zheng, Roman Fischer, et al.
Molecular Therapy : the Journal of the American Society of Gene Therapy|December 12, 2025
Targeted BDNF upregulation via upstream open reading frame disruptionNing Feng, Thomas Goedert, Nenad Svrzikapa, et al.
Nature Reviews. Drug Discovery|December 15, 2019
Therapies for rare diseases: therapeutic modalities, progress and challenges aheadErik Tambuyzer, Benjamin Vandendriessche, Christopher P Austin, et al.
Nature Reviews. Drug Discovery|January 9, 2020
Publisher Correction: Therapies for rare diseases: therapeutic modalities, progress and challenges aheadErik Tambuyzer, Benjamin Vandendriessche, Christopher P Austin, et al.
Amyotrophic Lateral Sclerosis & Frontotemporal Degeneration|March 11, 2024
Dyspnea (breathlessness) in amyotrophic lateral sclerosis/motor neuron disease: prevalence, progression, severity, and correlatesCarolyn A Young, Amina Chaouch, Christopher J Mcdermott, et al.
Annals of Neurology|November 16, 2024
Elevated Cerebrospinal Fluid Ubiquitin Carboxyl-Terminal Hydrolase Isozyme L1 in Asymptomatic C9orf72 Hexanucleotide Repeat Expansion CarriersElizabeth R Dellar, Iolanda Vendrell, Benazir Amein, et al.
Nature Communications|November 26, 2021
Axonal TDP-43 condensates drive neuromuscular junction disruption through inhibition of local synthesis of nuclear encoded mitochondrial proteinsTopaz Altman, Ariel Ionescu, Amjad Ibraheem, et al.
Human Mutation|October 20, 2011
A recurrent loss-of-function alanyl-tRNA synthetase (AARS) mutation in patients with Charcot-Marie-Tooth disease type 2N (CMT2N)Heather M McLaughlin, Reiko Sakaguchi, William Giblin, et al.
Human Brain Mapping|October 3, 2025
Divergent Brain Network Activity in Asymptomatic C9orf72 and SOD1 Variant Carriers Compared With Established Amyotrophic Lateral SclerosisMichael Trubshaw, Chetan Gohil, Evan Edmond, et al.
Neurobiology of Disease|August 18, 2020
An ALS-linked mutation in TDP-43 disrupts normal protein interactions in the motor neuron response to oxidative stressEmily Feneberg, David Gordon, Alexander G Thompson, et al.
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