Results 51 to 60 of about 1,467,614 (286)

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

SPG4 and Dementia: Expanding the Clinical Spectrum

Annals of Clinical and Translational Neurology, EarlyView.
ABSTRACT Objective Hereditary spastic paraplegia (HSP) is a group of disorders characterized by progressive spasticity and lower limb weakness, with mutations in SPG4/SPAST being the most common cause. Detailed studies and clinical and molecular comparisons across different populations are missing.
Emanuele Panza, Arun Meyyazhagan, Eliseo Picchi, Gustavo Ribas, Ingrid Faber, Ryosuke Miyamoto, Preethi Basavaraju, Paolo Eusebi, Haripriya Kuchi Bhotla, Mario Stasi, Fabrizio Gaudiello, Francesco Patti, Filippo Maria Santorelli, Marcondes Cavalcante França Jr, José Luiz Pedroso, Orlando Graziani Povoas Barsottini, Hélio Afonso Ghizoni Teive, Peter Henry St George‐Hyslop, Toshitaka Kawarai, Antonio Orlacchio   +19 more
wiley   +1 more source

Folklore argentino: aspectos introductorios, definiciones y debates

Arte e Investigación, 2013
La construcción de la categoría folklore se ha caracterizado por incluir una serie de contradicciones y de paradojas, que involucran conceptualizaciones y reflexiones letradas respecto de la cultura popular, además de las disputas ideológicas, políticas ...
Santiago Romé
doaj  

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

Spin and Rotation in General Relativity [PDF]

, 2001
Rapporteur's Introduction to the GT8 session of the Ninth Marcel Grossmann Meeting (Rome, 2000); to appear in the Proceedings.Comment: LaTeX file, no figures, 15 ...
E.P. Wigner, F. Gürsey, F.W. Hehl, J.D. Bjorken, L.H. Ryder, L.L. Foldy, W. Pauli   +6 more
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Sarilumab in Polyarticular‐Course Juvenile Idiopathic Arthritis: Dose‐Finding and 1‐Year Analysis of a Phase 2b, Open‐Label, Multicenter Study

Arthritis Care &Research, EarlyView.
Objective This study assessed sarilumab in treating patients with polyarticular‐course juvenile idiopathic arthritis (pcJIA). Methods This phase 2b, open‐label study (NCT02776735) consisted of three sequential parts (each with a core‐treatment and extension phase).
Fabrizio De Benedetti, Inmaculada C. Penadés, Irina Nikishina, Ivan Foeldvari, Alberto J. Spindler, Anna Kozlova, Nadina Rubio‐Pérez, Pierre Quartier, Zbigniew Żuber, Raul Barria, Daniel Clemente, Gabriel V. Cornejo, Katherine Marzan, Nancy Liu, Christine Xu, Stephen DiMartino, Angeliki Giannelou, Fei Cao‐Ghoul, Bolanle Akinlade, Lydie Baret‐Cormel   +19 more
wiley   +1 more source

Mycophenolate Mofetil Treatment Reduces the Risk of Treatment Escalation Due to Vascular Complications in Limited Cutaneous Systemic Sclerosis: Emulation of a Target Trial From the Italian Rheumatology Society SPRING Registry

Arthritis Care &Research, EarlyView.
Objective Mycophenolate mofetil (MMF) use in limited cutaneous systemic sclerosis (lcSSc) is relatively uncommon because of the lower fibrotic burden and the predominance of vascular complications. In vitro observations and clinical data from transplanted patients suggest a protective effect of MMF on endothelial function.
Enrico De Lorenzis, Gerlando Natalello, Rossella De Angelis, Lucrezia Verardi, Dilia Giuggioli, Gianluigi Bajocchi, Lorenzo Dagna, Silvia Bellando‐Randone, Giovanni Zanframundo, Rosario Foti, Fabio Cacciapaglia, Giovanna Cuomo, Alarico Ariani, Edoardo Rosato, Gemma Lepri, Francesco Girelli, Valeria Riccieri, Elisabetta Zanatta, Ilaria Cavazzana, Francesca Ingegnoli, Maria De Santis, Giuseppe Murdaca, Giuseppina Abignano, Giorgio Pettiti, Alessandra Della Rossa, Maurizio Caminiti, Annamaria Iuliano, Giovanni Ciano, Lorenzo Beretta, Gianluca Bagnato, Ennio Lubrano, Maria Ilenia De Andres, Alessandro Giollo, Cosimo Bruni, Martina Orlandi, Marco Fornaro, Marta Saracco, Cecilia Agnes, Pier Giacomo Cerasuolo, Gabriella Alonzi, Edoardo Cipolletta, Federica Lumetti, Amelia Spinella, Luca Magnani, Corrado Campochiaro, Giacomo De Luca, Veronica Codullo, Elisa Visalli, Carlo Iandoli, Antonietta Gigante, Greta Pellegrino, Erika Pigatto, Maria‐Grazia Lazzaroni, Franco Franceschini, Elena Generali, Gianna Mennillo, Simone Barsotti, Giuseppa Pagano Mariano, Federica Furini, Licia Vultaggio, Simone Parisi, Clara Lisa Peroni, Gerolamo Bianchi, Enrico Fusaro, Gian Domenico Sebastiani, Marcello Govoni, Salvatore D'Angelo, Franco Cozzi, Fabrizio Conti, Serena Guiducci, Andrea Doria, Carlo Salvarani, Florenzo Iannone, Maria Antonietta D'Agostino, Clodoveo Ferri, Marco Matucci Cerinic, Silvia Laura Bosello, on behalf of the SPRING Italian Registry Collaborators   +77 more
wiley   +1 more source

Kyzikos Kazısı 2022 Yılı Sikke Buluntuları

Journal of Literature and Humanities
Antik Dönem’de Mysia Bölgesi sınırları içerisinde yer alan Kyzikos günümüzde Balıkesir ili Erdek ilçesinin Düzler Mahallesi’nde yer almaktadır. Kent, Miletoslular tarafından ilk MÖ 756’da ikinci kez de MÖ 679 yılında kurulmuştur. Kyzikos kazılarının 2022
Mehmet Kayhan Murat
doaj   +1 more source

Public Communication and the Barriers to Participation: The Case of Rome from an Open Government Perspective

Partecipazione e Conflitto, 2020
The City of Rome inaugurated its open government programmes in 2016, but its heuristic relevance has been understudied. By analysing this peculiar case, this article proposes a reflection on the challenges of implementing open government in local ...
Emiliana De Blasio, Cecilia Colasanti, Donatella Selva   +2 more
doaj   +1 more source

'The influence of geography on the development of early Rome' : a thesis submitted in partial fulfilment of the requirements for a Master of Arts in History; School of Humanities at Massey University, Manawatu, New Zealand [PDF]

, 2018
Geography was an influential factor in the development of the site and city of Rome, affecting both the city itself, and the wider region. Rome was situated in a favorable position on the western cost of the Italian peninsula, with a rich hinterland ...
Putt, Matthew Karl
core