Results 231 to 240 of about 1,819,835 (332)

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

Federated Inverse Probability Treatment Weighting for Individual Treatment Effect Estimation. [PDF]

ACM Trans Intell Syst Technol
Yin C, Chen HY, Chao WL, Zhang P.
europepmc   +1 more source

Quantifying the Impact of Ocrelizumab on Paramagnetic Rim Lesions in Multiple Sclerosis

Annals of Clinical and Translational Neurology, EarlyView.
ABSTRACT Paramagnetic rim lesions (PRLs) are a subset of chronic active multiple sclerosis (MS) lesions marked by iron‐laden microglia and macrophages. Ocrelizumab, a monoclonal antibody targeting CD20+ B cells, suppresses acute MS activity, but its effect on PRLs remains unclear. In a longitudinal study of 29 ocrelizumab‐treated patients with at least
Kimberly H. Markowitz, Neha V. Safi, Iliana Pliska‐Bloch, Ulrike W. Kaunzner, Ha Luu, Mert Sisman, Yi Wang, Thanh D. Nguyen, Sandra Hurtado Rúa, Susan A. Gauthier   +9 more
wiley   +1 more source

Analytical and computational solution for the estimation of SNP-heritability in biobank-scale and distributed datasets. [PDF]

PLoS Comput Biol
Qi GA, Zhang QX, Kang J, Li T, Xu X, Zhang Z, Fan Z, Liu S, Chen GB.   +8 more
europepmc   +1 more source

Clinical Validation of Plasma p‐217tau in Neurological Diseases

Annals of Clinical and Translational Neurology, EarlyView.
ABSTRACT Objective Plasma p‐217tau is a minimally invasive but specific biomarker for diagnosing Alzheimer's disease (AD). However, its disease specificity remains to be clinically evaluated. We validated the reliability of the p‐217tau biomarker in 12 other neurological diseases.
Takeshi Kawarabayashi, Takumi Nakamura, Ryoma Takahashi, Tetsuya Ueda, Seiji Kinoshita, Chikage Uchida, Takashi Sugawara, Kentaro Hashimoto, Kunihiko Ishizawa, Masakuni Amari, Hiroo Kasahara, Yoshio Ikeda, Masamitsu Takatama, Mikio Shoji   +13 more
wiley   +1 more source

Deep Robust Moving Horizon Estimation for Nonlinear Multi-Rate Systems. [PDF]

Sensors (Basel)
Wang R, Wen S, Chen B.
europepmc   +1 more source

From noise to models to numbers: Evaluating negative binomial models and parameter estimations in single-cell RNA-seq. [PDF]

PLoS Comput Biol
Wang Y, Shu Z, Cao Z, Grima R.
europepmc   +1 more source

Distributed fixed time adaptive cooperative tracking control for quadrotor unmanned aerial vehicle. [PDF]

Sci Rep
Zhou J, Liang X, Shi M, Liu Z.
europepmc   +1 more source

Design and Sensing Frameworks of Soft Octopus-Inspired Grippers Toward Artificial Intelligence. [PDF]

Biomimetics (Basel)
Choi S, Jang J, Lee J, Kim DW.
europepmc   +1 more source