ACMG-AMP American College of Medical Genetics and Genomics–Association for Molecular Pathology ALS amyotrophic lateral sclerosis ATPase adenosine triphosphatase CADD combined annotation-dependent depletion DNA deoxyribonucleic acid EMG electromyography H&E hematoxylin and eosin hnRNPA1 heterogeneous nuclear ribonucleoprotein A1 IRB Institutional Review Board KD knockdown MSP multisystem proteinopathy NRES National Research Ethics Service RBP RNA-binding protein RRM RNA recognition motif WES...