Results 151 to 160 of about 139,916 (258)

Ofatumumab in Myelin Oligodendrocyte Glycoprotein Antibody–Associated Disease: A Comparison With Rituximab

Annals of Clinical and Translational Neurology, EarlyView.
ABSTRACT Objective To evaluate the efficacy and safety of ofatumumab in patients with myelin oligodendrocyte glycoprotein antibody–associated disease (MOGAD), and compare it with rituximab. Methods We conducted a single–center, observational study including 22 MOGAD patients treated with ofatumumab and 21 treated with rituximab.
Yuxin Fan, Lei Zhou, Jingzi ZhangBao, Hongmei Tan, Zhouzhou Wang, Chao Quan   +5 more
wiley   +1 more source

A repeat expansion in GOLGA8A is a major risk factor for atypical frontotemporal lobar degeneration with ubiquitin-positive inclusions. [PDF]

Nat Genet
De Coster W, Van den Broeck M, Baker M, Ghayal NB, Wynants S, Batzler A, Pottier C, Alidadiani S, Küçükali F, Jenkins GD, Policarpo R, van Blitterswijk M, DeJesus-Hernandez M, Soto-Beasley AI, Faura J, Coopman E, Hutten S, Mol MO, Wallon D, Sieben A, Finger EC, Murray ME, Forrest SL, Tartaglia MC, Troakes C, van Rooij JGJ, Nguyen AT, Reichard RR, Woodman NL, Nana AL, Weintraub S, Gefen T, De Vil B, Bodi I, Lopez OL, Boluda S, Belliard S, Lebert F, Marguet F, Mao Q, Mesulam MM, Boxer AL, Vandenbulcke M, Suh E, Schaeverbeke J, Lambert JC, Scholz SW, Dalgard CL, Traynor BJ, Gibbs RJ, Schellenberg GD, Dormann D, Joris G, De Pooter T, De Rijk P, D'Hert S, Van Dongen J, van der Zee J, Strazisar M, Gearing M, Kukar T, Flanagan M, Engelborghs S, Ghetti B, Newell KL, King A, Roeber S, Rosen HJ, Spina S, Cras P, Ertekin-Taner N, Wszolek ZK, Uitti RJ, Cheshire WP, Singer W, Herms J, Josephs KA, Whitwell JL, Petersen RC, Pasquier F, Nicolas G, Castellani R, Glass J, Miller BL, Kovacs GG, Rissman RA, Hiniker A, Deramecourt V, Ang LC, Lee-Way J, Van Deerlin VM, Dugger BN, Thal DR, Grinberg LT, Cruchaga C, Arzberger T, Munoz DG, Keith J, Zinman L, Rogaeva E, Lee EB, Haggarty SJ, Ansorge O, Husain M, Halliday GM, Al-Sarraj S, Ross OA, Sleegers K, Vandenberghe R, Boeve BF, Graff-Radford NR, Kofler J, White CL, Lashley T, Neumann M, Biernacka JM, Seeley WW, Seelaar H, van Swieten JC, Rohrer JD, Dickson DW, Mackenzie IRA, Rademakers R.   +122 more
europepmc   +1 more source

Dimethyl Fumarate, But Not Rituximab, Reduces Serum GFAP Levels and PIRMA in Relapsing–Remitting MS

Annals of Clinical and Translational Neurology, EarlyView.
ABSTRACT Objective Serum neurofilament light chain (sNfL) and glial fibrillary acidic protein (sGFAP) levels are believed to reflect mainly acute and chronic disease processes in multiple sclerosis (MS), respectively. In this study, we investigated whether dimethyl fumarate (DMF) and rituximab (RTX) differentially affect these biomarkers.
F. Shawket, J. Lycke, J. Salzer, F. Piehl, K. Fink, N. Lange, J. Mellergård, C. Malmeström, P. Sundström, I. Erngren, A. al‐Grety, E. Freyhult, K. Kultima, J. Burman, A. Svenningsson   +14 more
wiley   +1 more source

RFC1 Repeat Expansion in Chronic Cough: Findings From the Korean Chronic Cough Registry. [PDF]

Allergy Asthma Immunol Res
Park KE, Lee J, Choi JP, Oh JY, Won HK, Lee HY, Birring SS, Park HW, Cho SH, Song WJ.   +9 more
europepmc   +1 more source

Safety and Tolerability of Givinostat: Evidence From Real‐World and Clinical Practice

Annals of Clinical and Translational Neurology, EarlyView.
ABSTRACT Objective The aim of our study was to establish the prevalence of adverse events in a real‐world setting in boys living with Duchenne muscular dystrophy (DMD) treated with givinostat as part of an Expanded Access Program (EAP) in Italy. Methods The cohort included 90 ambulant boys, with age when treatment started between 6 and 23 years (mean ...
Marika Pane, Anna Capasso, Chiara Arpaia, Adele D’Amico, Emilio Albamonte, Federica Trucco, Maria Sframeli, Riccardo Masson, Francesca Magri, Luca Bello, Romina Venditti, Claudia Dosi, Michela Catteruccia, Michele Tosi, Claudio Bruno, Sonia Messina, Giacomo Comi, Elena Pegoraro, Valeria A. Sansone, Eugenio Mercuri   +19 more
wiley   +1 more source

Aberrant splicing exonizes C9orf72 repeat expansion in ALS/FTD. [PDF]

Nat Neurosci
Yang S, Wijegunawardana D, Sheth U, Veire AM, Salgado JMS, Arab T, Agrawal M, Zhou J, Pereira JD, Gendron TF, Guo JU.   +10 more
europepmc   +1 more source

Repeat Expansion Disorders: Bridging the Gap

Clinical Chemistry, 2022
openaire   +2 more sources

Early Clinical, Imaging, and Pathological Characteristics of SRPK3/TTN‐Digenic Myopathy

Annals of Clinical and Translational Neurology, EarlyView.
ABSTRACT Objective SRPK3/TTN‐digenic myopathy was recently established as a skeletal muscle myopathy caused by digenic inheritance. This study characterizes the early clinical presentation of SRPK3/TTN‐digenic myopathy in one previously reported and seven newly identified pediatric patients.
Rotem Orbach, Sandra Donkervoort, Carola Hedberg‐Oldfors, Giovanni Baranello, Dimah Saade, Precilla D'Souza, Ruchee Patel, Eva Michael, A. Reghan Foley, Diana Bharucha‐Goebel, S. Jin Haugland, Meghan McAnally, Omer Abdul Hamid, Katherine Chao, Ellen F. Macnamara, Alan H. Beggs, Anna Sarkozy, Juliane Mueller, Steven A. Moore, Richard S. Finkel, Cynthia J. Tifft, Francesco Muntoni, Anders Oldfors, Carsten G. Bönnemann   +23 more
wiley   +1 more source

Compound Heterozygote Friedreich Ataxia Patients With Covert Proximal FXN Gene Deletions

Annals of Clinical and Translational Neurology, EarlyView.
ABSTRACT We present Friedreich ataxia patients with frataxin gene deletions. Data and records were collected at the Children's Hospital of Philadelphia from patients enrolled in the FACOMS natural history study. Patients with proximal deletions initially diagnosed with only one GAA expanded allele had more severe disease than their homozygous expansion
Michael P. Lazaropoulos, Morgan C. Devore, Christina Lam, Courtney Park, Sanjay Bidichandani, David R. Lynch   +5 more
wiley   +1 more source

Comparative Effectiveness and Safety of Inebilizumab Versus Rituximab in AQP4‐IgG‐Positive NMOSD

Annals of Clinical and Translational Neurology, EarlyView.
ABSTRACT Objective Rituximab (anti‐CD20, RTX) and inebilizumab (anti‐CD19, INE) represent B‐cell‐depleting therapies used for aquaporin‐4 antibody‐positive (AQP4‐IgG+) neuromyelitis optica spectrum disorder (NMOSD); however, direct comparative evidence remains limited.
Jie Lin, Binbin Xue, Dewei Xie, Jia Li, Juyuan Pan, Lanbing Zhu, Qiaowen Tong, Jing Lin, Ruofan Zhu, Xu Zhang, Junhui Xia, Xiang Li   +11 more
wiley   +1 more source