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Human Molecular Genetics|August 14, 2010
SMN deficiency disrupts brain development in a mouse model of severe spinal muscular atrophyThomas M Wishart, Jack P-W Huang, Lyndsay M Murray, et al.
Ebiomedicine|August 13, 2022
Targeting phosphoglycerate kinase 1 with terazosin improves motor neuron phenotypes in multiple models of amyotrophic lateral sclerosisHelena Chaytow, Emily Carroll, David Gordon, et al.
EMBO Molecular Medicine|September 19, 2023
PRMT inhibitor promotes SMN2 exon 7 inclusion and synergizes with nusinersen to rescue SMA miceAnna J Kordala, Jessica Stoodley, Nina Ahlskog, et al.
Plos Genetics|December 7, 2011
Charcot-Marie-Tooth-linked mutant GARS is toxic to peripheral neurons independent of wild-type GARS levelsWilliam W Motley, Kevin L Seburn, Mir Hussain Nawaz, et al.
Human Brain Mapping|January 4, 2008
Investigation of white matter pathology in ALS and PLS using tract-based spatial statisticsOlga Ciccarelli, Timothy E Behrens, Heidi Johansen-Berg, et al.
Proceedings of the National Academy of Sciences of the United States of America|September 14, 2016
Systemic peptide-mediated oligonucleotide therapy improves long-term survival in spinal muscular atrophySuzan M Hammond, Gareth Hazell, Fazel Shabanpoor, et al.
Amyotrophic Lateral Sclerosis & Frontotemporal Degeneration|July 25, 2022
Measuring disability in amyotrophic lateral sclerosis/motor neuron disease: the WHODAS 2.0-36, WHODAS 2.0-32, and WHODAS 2.0-12Carolyn A Young, John Ealing, Christopher J McDermott, et al.
Human Molecular Genetics|August 16, 2011
Reversible molecular pathology of skeletal muscle in spinal muscular atrophyChantal A Mutsaers, Thomas M Wishart, Douglas J Lamont, et al.
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.
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