Results 151 to 160 of about 16,606 (215)

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

The structural, functional, and therapeutic potential of metacaspases in fungi and protozoa. [PDF]

FEBS J
Duarte ACM, Trujilho MNR, Valdivia KSSM, Araujo AF, de Lorena LMC, de Oliveira VH, Pereira BV, Leme ER, Júdice WAS, Machado MFM.   +9 more
europepmc   +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

Engineering the Thermometric Response of Dinuclear Eu^III Complexes via Terminal Ligand-Induced Nonradiative Processes. [PDF]

ACS Omega
Beltrame ACF, Silva RAN, Lima SAM, Marchiò L, Melegari M, Artizzu F, Bispo-Jr AG, Pires AM.   +7 more
europepmc   +1 more source

Characterizing Cutaneous α‐Synuclein Deposition and Seeding Activity in Parkinson's Disease Subtypes

Annals of Clinical and Translational Neurology, EarlyView.
ABSTRACT Objective Cutaneous phosphorylated α‐synuclein (p‐syn) and α‐synuclein seeding activity are promising biomarkers for Parkinson's disease (PD), but their clinical value remains uncertain due to disease heterogeneity. This study evaluates these two biomarkers in PD patients to inform phenotype‐specific diagnosis and disease severity assessment ...
Yuting Jin, Tianyuan Yang, Minglei Liu, Haiyang Luo, Jing Yuan, Ke Zhang, Xinsheng Han, Jing Yang, Yuming Xu   +8 more
wiley   +1 more source

Novel Biallelic PLEKHG5 Variant Associated With Intermediate Charcot-Marie-Tooth Disease: Case Report From South America. [PDF]

J Peripher Nerv Syst
Vidon RO, Tomaselli PJ, Bittar-Braune C, Pitta IJ, de Lacerda GCB, Jardim M.   +5 more
europepmc   +1 more source

Trajectories of Physical Function in Canadian Children With Juvenile Idiopathic Arthritis

Arthritis Care &Research, EarlyView.
Objective We describe trajectories of physical function in children newly diagnosed with juvenile idiopathic arthritis (JIA) and identify trajectories with persisting functional impairments and associated baseline characteristics. Methods We included patients enrolled in the Canadian Alliance of Pediatric Rheumatology Investigators (CAPRI) Registry ...
Clare Cunningham, Meghan McPherson, Lillian Lim, Roberta A. Berard, Matthew Berkowitz, Jean‐Philippe Proulx‐Gauthier, Brian M. Feldman, Nicole Johnson, Dax G. Rumsey, Heinrike Schmeling, Lori B. Tucker, Thomas Loughin, Kristin M. Houghton, Jaime Guzman, for the Canadian Alliance of Pediatric Rheumatology Investigators (CAPRI) Registry Investigators, David A. Cabral, Gaëlle Chédeville, Ciarán M. Duffy, Kerstin Gerhold, Jaime Guzman, Linda Hiraki, Adam M. Huber, Heinrike Schmeling, Natalie J. Shiff, Lori B. Tucker, Julie Barsalou, Daniah Basodan, Michelle Batthish, Roberta A. Berard, Nicholas Blanchette, Gilles Boire, Roxana Bolaria, Alessandra Bruns, David A. Cabral, Sarah Campillo, Tania Cellucci, Mercedes Chan, Gaëlle Chédeville, Amieleena Chhabra, Julie Couture, Paul Dancey, Jean‐Jacques De Bruycker, Erkan Demirkaya, Muhammed Dhalla, Ciarán M. Duffy, Talal El Tal, Tommy Gerschman, Jaime Guzman, Liane Heale, Julie Herrington, Kristin M. Houghton, Adan M. Huber, Andrea Human, Nicole Johnson, Roman Jurencak, Bianca Lang, Claire LeBlanc, Lillian Lim, Lily S. H. Lim, Nadia Luca, Jeanine McColl, Tara McGrath, Tamara McMillan, Megan McPherson, Paivi Miettunen, Kimberly Morishita, Honyan Ng, Jonathan Park, Jean‐Philippe Proulx‐Gauthier, Johannes Roth, Evelyn Rozenblyum, Dax G. Rumsey, Heinrike Schmeling, Rosie Scuccimarri, Gordon Soon, Rebeka Stevenson, Elizabeth Stringer, Herman Tam, Lori B. Tucker, Marinka Twilt, Ruud H. J. Verstegen, Karen Watanabe Duffy   +81 more
wiley   +1 more source

Synthesis of <i>N</i>‑Substituted Acenaphtho[1,2‑<i>b</i>]pyrroles and Dibenzo[<i>e,g</i>]indoles with Promising Antileukemic Activity from Morita-Baylis-Hillman Adducts. [PDF]

ACS Omega
Arantes J, Rodrigues MT, Rosendo G, Porreca R, Vicari HP, Santos H, Machado-Neto JA, Coelho F.   +7 more
europepmc   +1 more source

Obesity Subtypes and Longitudinal Trajectories of Function Over Seven Years of Follow‐Up: Data From the Multicenter Osteoarthritis Study

Arthritis Care &Research, EarlyView.
Objective Obesity, defined by body mass index (BMI) ≥30 kg/m2, is a risk factor for functional limitations in people with knee osteoarthritis (OA). However, function varies among such individuals. Our objective was to evaluate the implications of obesity subtypes on longitudinal patterns of physical functioning in people with or at risk for knee OA ...
Kristine Godziuk, Sarah Tilley, Michael LaValley, Michael C. Nevitt, Cora E. Lewis, James C. Torner, Tuhina Neogi, for the Multicenter Osteoarthritis Study Group   +7 more
wiley   +1 more source

Reduced handgrip strength and body mass index, gender, and diagnosis are associated with length of stay in hospitalized patients. [PDF]

Arq Bras Cir Dig
Mazzini LR, Leandro-Merhi VA.
europepmc   +1 more source