Results 191 to 200 of about 4,157,307 (334)

Onasemnogene Abeparvovec in Type I Spinal Muscular Atrophy: 24‐Month Follow‐Up From the Italian Registry

Annals of Clinical and Translational Neurology, EarlyView.
ABSTRACT Objective Onasemnogene abeparvovec (OA) is an AAV9‐based gene therapy for spinal muscular atrophy type I (SMA I). Real‐world outcomes show increased response variability compared to clinical trials, and follow‐up data beyond 12–18 months are limited.
Marika Pane, Giorgia Coratti, Chiara Cutrì, Antonio Varone, Riccardo Masson, Adele D'Amico, Valeria Sansone, Sonia Messina, Federica Ricci, Chiara Ticci, Claudio Bruno, Caterina Agosto, Francesca Benedetti, Antonella Pini, Sabrina Siliquini, Massimiliano Filosto, Alberto Zambon, Ilaria Bitetti, Maria Rosaria Manna, Claudia Dosi, Riccardo Zanin, Stefano Parravicini, Roberto De Sanctis, Giulia Stanca, Michela Catteruccia, Michele Tosi, Irene Mizzoni, Emilio Albamonte, Valentina Franchino, Maria Sframeli, Ilaria Cavallina, Elena Procopio, Michele Sacchini, Simone Morando, Noemi Brolatti, Federica Trucco, Gaia Scarpini, Elena Briganti, Beatrice Berti, Concetta Palermo, Daniela Leone, Stefano C. Previtali, Eugenio Mercuri, the ITASMAC working group   +43 more
wiley   +1 more source

Minimally Clinically Important Difference of the Clinical Assessment Scale in Autoimmune Encephalitis

Annals of Clinical and Translational Neurology, EarlyView.
ABSTRACT The Clinical Assessment Scale in Autoimmune Encephalitis (CASE) tracks disease severity in autoimmune encephalitis (AE), but no threshold for significant change exists. We aimed to determine the minimally clinically important difference (MCID) for CASE.
Yihui Goh, Yoon Hee Shin, Soo Hyun Ahn, Su Yee Mon, Soo Jean Shin, Woo‐Jin Lee, Yoonhyuk Jang, Myoung‐Jin Jang, Soon‐Tae Lee   +8 more
wiley   +1 more source

Viral evolution during primary infection in immunocompromised hosts. [PDF]

PLoS Comput Biol
Craig M, Deng X, McLeod DV.
europepmc   +1 more source

Viral evolution of SIV chimpanzee toward HIV-1 using humanized mice. [PDF]

J Med Primatol, 2023
Curlin JZ, Schmitt K, Remling-Mulder L, Tibbitts CV, O'Connor S, Marx P, Akkina R.   +6 more
europepmc   +1 more source

ALDOA Promotes Glycolysis and NLRP3/GSDMD Pyroptosis to Accelerate ALS Progression

Annals of Clinical and Translational Neurology, EarlyView.
ABSTRACT Objective Amyotrophic lateral sclerosis (ALS) is characterized by progressive motor neuron degeneration. Glycolytic dysregulation is implicated in disease progression, yet the underlying mechanisms remain unclear. This study investigates how Aldolase A (ALDOA) drives ALS progression through glycolysis‐mediated motor neuron pyroptosis.
Kaixin Yan, Yan Jiang, Yuxuan Yong, Tianshuo Zhang, Niannian Zhang, Qianqian Zeng, Xue Gong, Li Meng, Fangfang Bi, Yongmin Liu   +9 more
wiley   +1 more source

The mutational landscape of SARS-CoV-2 provides new insight into viral evolution and fitness. [PDF]

Nat Commun
Symons J, Chung C, Verheijen BM, Shemtov SJ, de Jong D, Amatngalim G, Nijhuis M, Vermulst M, Gout JF.   +8 more
europepmc   +2 more sources

Chronic infections can generate SARS-CoV-2-like bursts of viral evolution without epistasis. [PDF]

Virus Evol
Rodríguez-Horta E, Strahan J, Dinner AR, Barton JP.   +3 more
europepmc   +1 more source

Pharmacokinetics, Pharmacodynamics, and Safety of Subcutaneous Belimumab in Pediatric Patients With Systemic Lupus Erythematosus: A Multicenter, Open‐Label Trial

Arthritis Care &Research, EarlyView.
Objective This study aimed to characterize the pharmacokinetics, pharmacodynamics, safety, and exploratory efficacy of subcutaneous belimumab in pediatric patients with active systemic lupus erythematosus (SLE) receiving standard therapy. Methods This single‐arm, multicenter, open‐label trial (GSK study 200908; ClinicalTrials.gov identifier ...
Hermine I. Brunner, Jordi Antón, Inmaculada Calvo‐Penadés, Richard Dimelow, Gerd Horneff, Sylvia Kamphuis, Rebecca Marino, Andre van Maurik, Kirsten Minden, Masaaki Mori, Josephine Ocran‐Appiah, Christel Wilkinson, Yuichi Yamasaki, Deepak Assudani, for the Pediatric Rheumatology Collaborative Study Group (PRCSG) and Paediatric Rheumatology International Trials Organisation (PRINTO) Network investigators   +14 more
wiley   +1 more source

A combination of broadly neutralizing antibodies prevents in vitro SHIV162p3 cell-free transmission more efficiently than cell to cell transmission

Journal of Virus Eradication, 2016
K. Suphaphiphat, M. Tolazzi, G. Scarlatti, R. Legrand, M. Cavarelli   +4 more
doaj   +1 more source

Viro-immunological evaluation in an immunocompromised patient with long-lasting SARS-CoV-2 infection

Emerging Microbes and Infections, 2022
A. Mancon, A. Rizzo, D. Mileto, S. Grosso, A. Foschi, M. Cutrera, A. Capetti, I. Faggion, A. Anselmo, A. Monte, S. Fillo, G. Rizzardini, M. R. Gismondo, V. Micheli   +13 more
doaj   +1 more source