Results 131 to 140 of about 1,397,252 (312)

Onasemnogene Abeparvovec in Patients With SMA: Interim Results of the RESTORE Registry in Japan

Annals of Clinical and Translational Neurology, EarlyView.
ABSTRACT Objective There are limited real‐world data regarding the safety and effectiveness of onasemnogene abeparvovec (OA; Zolgensma) infusion, a one‐time gene replacement therapy, for Japanese patients with spinal muscular atrophy (SMA). We aimed to improve understanding of the real‐world outcomes for OA in Japan.
Kayoko Saito, Kamal Benguerba, Ken Tsuchida, Kazushige Yazawa, Isao Tsumiyama, Hiromitsu Kayama, Sandra P. Reyna, Farid Khan, Richard S. Finkel   +8 more
wiley   +1 more source

Behavioural simulation of biological neuron systems using VHDL and VHDL-AMS

, 2007
The investigation of neuron structures is an incredibly difficult and complex task that yields relatively low rewards in terms of information from biological forms (either animals or tissue).
Brown, Andrew D., Wilson, Peter R., Chad, John, Bailey, Julian   +3 more
core   +1 more source

White Matter Microstructural Abnormalities in Neonatal Onset Genetic Epilepsy

Annals of Clinical and Translational Neurology, EarlyView.
ABSTRACT Objective Recent evidence indicates that epilepsy is associated with abnormal white matter. If seizures alter white matter, then the impact upon network function, epileptogenesis, and cognition could be pronounced in neonates undergoing rapid developmental myelination. Neonates with epilepsy due to nonstructural genetic causes provide a unique
Amanda G. Sandoval Karamian, Tianjia Zhu, Hao Huang, Shavonne L. Massey, Mark P. Fitzgerald, Darshana Parikh, Arastoo Vossough, Ingo Helbig, Nicholas S. Abend   +8 more
wiley   +1 more source

Neurochemical Endpoints to Inform Early‐Stage Trials of Spinocerebellar Ataxia 2 and 3 in a Multisite Setting

Annals of Clinical and Translational Neurology, EarlyView.
ABSTRACT Objective Neurochemical levels measured by brain MR spectroscopy (MRS) have been proposed as endpoints for clinical trials in early‐stage spinocerebellar ataxia (SCA) trials. We tested their trial‐readiness by quantifying neurochemicals in three affected brain regions in early‐stage cohorts of SCA2 and SCA3, examining their reproducibility in ...
James M. Joers, Yaqiao Wei, Dinesh K. Deelchand, Adam Berrington, Young Woo Park, Guita Banan, Michal Povazan, Jens T. Rosenberg, André J. W. van der Kouwe, Tetsuo Ashizawa, Khalaf O. Bushara, Chiadi U. Onyike, Sub H. Subramony, Jeremy D. Schmahmann, Brian J. Soher, Peter B. Barker, Thomas H. Mareci, Eva‐Maria Ratai, Lynn E. Eberly, Gülin Öz   +19 more
wiley   +1 more source

T1 Over Squared Proton Density Ratio to Characterize Multiple Sclerosis Lesions

Annals of Clinical and Translational Neurology, EarlyView.
ABSTRACT Objective Differentiating remyelinated from demyelinated lesions in MS remains challenging without histological confirmation. This study introduces the T1‐to‐PD2 ratio (TPR) imaging approach and evaluates its ability to characterize MS lesions alongside other quantitative MRI (qMRI) metrics. Methods Thirty individuals with MS (mean age: 47.5 ± 
Sarah J. Wright, Scotty G. McKay, Jean Ramos Bou, Fen Bao, Vivian B. Truong, Chenyang Li, Li Jiang, Anza B. Memon, E. Mark Haacke, Yulin Ge, Yongsheng Chen   +10 more
wiley   +1 more source

Biomarkers of disease in a case of familial lower motor neuron ALS

, 2010
ALS is a fatal disease with variable clinical course. There is no single reliable marker of disease progression. Sufficient records were available to study the case history of four family members with the uncommon G93V SOD1 mutation.
Baumann, Fusun, Pettitt, Anthony, Nicholson, Garth, Hutchinson, Nicole, Pannek, Kerstin, Nicholson, Garth A., Rose, Stephen E., McCombe, Pamela A., Henderson, Robert, Henderson, Robert D., Pettitt, Tony, Rose, Stephen, McCombe, Pamela, Nicole Hutchinson, Garth A. Nicholson, Fusun Baumann, Kerstin Pannek, Anthony Pettitt, Stephen E. Rose, Robert D. Henderson, Pamela A. Mccombe   +20 more
core   +1 more source

Tofersen Treatment in

Mayo Clinic Proceedings: Innovations, Quality & Outcomes
Objective: To evaluate the effectiveness of Tofersen in patients with superoxide dismutase 1 gene ( SOD1-ALS ) patients in France in a real-world setting, using disease progression within patient comparisons and with a historical cohort.
Daniells Erazo, MD, PhD, Adele Hesters, MD, Gaëlle Bruneteau, MD, PhD, Elisa De La Cruz, MD, Emilien Bernard, MD, Aude-Marie Grapperon, MD, Gwendal Le Masson, MD, PhD, Pascal Cintas, MD, Marie-Hélène Soriani, MD, Nathalie Guy, MD, Julien Cassereau, MD, PhD, Jean-Philippe Camdessanché, MD, PhD, Steeve Genestet, MD, PhD, Philippe Corcia, MD, PhD, Sophie Pittion, MD, Véronique Danel, MD, Mathilde Filliatre, MD, Ariane Choumert, MD, Agnes Jacquin-Piques, MD, François Salachas, MD, Florence Esselin, MD, Adrien Bohic, MD, Géraldine Lautrette, MD, Shahram Attarian, MD, PhD, Julie Catteau, PhD, Claire Guissart, MD, Marie-Claire Vincent, MD, Kevin Mouzat, MD, PhD, Marie Temple, MD, Patrick Vourc’h, MD, PhD, Philippe Couratier, MD, PhD   +30 more
doaj   +1 more source

Cognitive and Neuroimaging Divergence Between Juvenile and Adult FUS Amyotrophic Lateral Sclerosis

Annals of Clinical and Translational Neurology, EarlyView.
ABSTRACT Objective Amyotrophic lateral sclerosis (ALS) is a neurodegenerative disorder characterized by progressive motor neuron degeneration. Fused in sarcoma (FUS)‐associated juvenile ALS (jALS) represents a distinct and aggressive subgroup with rapid deterioration and poor prognosis.
Alexandra V. Jürs, Marcel Naumann, Annaliis Lehto, Hanna Schön, Jens Kurth, Johannes Prudlo, Andreas Hermann, Elisabeth Kasper   +7 more
wiley   +1 more source

Motor neuron disease [PDF]

Postgraduate Medical Journal, 1992
openaire   +2 more sources

Types of Motor Neuron Diseases

, 2017
Disorders of lower motor neurons (LMNs, or anterior horn cells) and upper motor neurons (UMNs), jointly termed motor neuron disorders (MNDs), are diverse and numerous.
Erik P Pioro, Nimish Thakore
core   +1 more source