Zobrazeno 1 - 10
of 120
pro vyhledávání: '"Scarpa A"'
Autor:
Roberto Giugliani, Antonio Gonzalez-Meneses, Maurizio Scarpa, Barbara Burton, Raymond Wang, Esmeralda Martins, Esmeralda Oussoren, Julia B. Hennermann, Brigitte Chabrol, Christina L. Grant, Angela Sun, Consuelo Durand, Joel Hetzer, Betsy Malkus, Deborah Marsden, J. Lawrence Merritt II
Publikováno v:
Orphanet Journal of Rare Diseases, Vol 19, Iss 1, Pp 1-9 (2024)
Abstract Background Mucopolysaccharidosis VII (MPS VII) is an ultra-rare, autosomal recessive, debilitating, progressive lysosomal storage disease caused by reduced activity of β-glucuronidase (GUS) enzyme. Vestronidase alfa (recombinant human GUS)
Externí odkaz:
https://doaj.org/article/035cbead2c0241dba2d01dd40974329c
Autor:
Laura K. Godfrey, Jan Forster, Sven-Thorsten Liffers, Christopher Schröder, Johannes Köster, Leonie Henschel, Kerstin U. Ludwig, David Lähnemann, Marija Trajkovic-Arsic, Diana Behrens, Aldo Scarpa, Rita T. Lawlor, Kathrin E. Witzke, Barbara Sitek, Steven A. Johnsen, Sven Rahmann, Bernhard Horsthemke, Michael Zeschnigk, Jens T. Siveke
Publikováno v:
Clinical Epigenetics, Vol 16, Iss 1, Pp 1-22 (2024)
Abstract Background Pancreatic ductal adenocarcinoma (PDAC) is an aggressive cancer with poor prognosis. It is marked by extraordinary resistance to conventional therapies including chemotherapy and radiation, as well as to essentially all targeted t
Externí odkaz:
https://doaj.org/article/d600d9b4f409474cb363cc7b8d335cf3
Autor:
Melissa P. Wasserstein, Robin Lachmann, Carla Hollak, Antonio Barbato, Renata C. Gallagher, Roberto Giugliani, Norberto Bernardo Guelbert, Julia B. Hennermann, Takayuki Ikezoe, Olivier Lidove, Paulina Mabe, Eugen Mengel, Maurizio Scarpa, Ebubekir Senates, Michel Tchan, Jesus Villarrubia, Beth L. Thurberg, Abhimanyu Yarramaneni, Nicole M. Armstrong, Yong Kim, Monica Kumar
Publikováno v:
Orphanet Journal of Rare Diseases, Vol 18, Iss 1, Pp 1-12 (2023)
Abstract Background Olipudase alfa is a recombinant human acid sphingomyelinase enzyme replacement therapy for non-central-nervous-system manifestations of acid sphingomyelinase deficiency (ASMD). The ASCEND randomized placebo-controlled trial in adu
Externí odkaz:
https://doaj.org/article/7737861ecbf54c38965ac8ebac64bc3c
Autor:
Quan Zheng, Jiajia Tang, Alexandra Aicher, Tony Bou Kheir, Berina Sabanovic, Preeta Ananthanarayanan, Chiara Reina, Minchun Chen, Jian-Min Gu, Bin He, Sonia Alcala, Diana Behrens, Rita T. Lawlo, Aldo Scarpa, Manuel Hidalgo, Bruno Sainz, Patricia Sancho, Christopher Heeschen
Publikováno v:
Journal of Experimental & Clinical Cancer Research, Vol 42, Iss 1, Pp 1-21 (2023)
Abstract Background Pancreatic ductal adenocarcinoma (PDAC) is a profoundly aggressive and fatal cancer. One of the key factors defining its aggressiveness and resilience against chemotherapy is the existence of cancer stem cells (CSCs). The importan
Externí odkaz:
https://doaj.org/article/2d7e2a41ca1e428c8e2dfa1def3561de
Autor:
Antonio Toscano, Olimpia Musumeci, Michele Sacchini, Sabrina Ravaglia, Gabriele Siciliano, Agata Fiumara, Elena Verrecchia, Melania Maione, Jennifer Gentile, Rita Fischetto, Grazia Crescimanno, Roberta Taurisano, Annalisa Sechi, Serena Gasperini, Vittoria Cianci, Lorenzo Maggi, Rossella Parini, Antonino Lupica, Maurizio Scarpa
Publikováno v:
Orphanet Journal of Rare Diseases, Vol 18, Iss 1, Pp 1-11 (2023)
Abstract Background The Italian Medicines Agency (AIFA) demands precise information on benefit/risk profile of home-based enzyme replacement therapy (ERT) for the treatment of patients with Pompe disease and Mucopolysaccharidosis type I (MPS I). This
Externí odkaz:
https://doaj.org/article/0a5becd85d924dc1b04b318ba6f147bc
Autor:
Margherita Piccardi, Manuel Gentiluomo, Stefania Bertoncini, Raffaele Pezzilli, Bálint Erőss, Stefania Bunduc, Faik G. Uzunoglu, Renata Talar-Wojnarowska, Tomas Vanagas, Cosimo Sperti, Martin Oliverius, Mateus Nóbrega Aoki, Stefano Ermini, Tamás Hussein, Ugo Boggi, Krzysztof Jamroziak, Evaristo Maiello, Luca Morelli, Ludmila Vodickova, Gregorio Di Franco, Stefano Landi, Andrea Szentesi, Martin Lovecek, Marta Puzzono, Francesca Tavano, Hanneke W. M. van Laarhoven, Alessandro Zerbi, Beatrice Mohelnikova-Duchonova, Hannah Stocker, Eithne Costello, Gabriele Capurso, Laura Ginocchi, Rita T. Lawlor, Giuseppe Vanella, Francesca Bazzocchi, Jakob R. Izbicki, Anna Latiano, Bas Bueno-de-Mesquita, Ruggero Ponz de Leon Pisani, Ben Schöttker, Pavel Soucek, Péter Hegyi, Maria Gazouli, Thilo Hackert, Juozas Kupcinskas, Lina Poskiene, Matteo Tacelli, Susanne Roth, Silvia Carrara, Francesco Perri, Viktor Hlavac, George E. Theodoropoulos, Olivier R. Busch, Andrea Mambrini, Casper H. J. van Eijck, Paolo Arcidiacono, Aldo Scarpa, Claudio Pasquali, Daniela Basso, Maurizio Lucchesi, Anna Caterina Milanetto, John P. Neoptolemos, Giulia Martina Cavestro, Dainius Janciauskas, Xuechen Chen, Roger Chammas, Mara Goetz, Hermann Brenner, Livia Archibugi, Michael Dannemann, Federico Canzian, Sergio Tofanelli, Daniele Campa
Publikováno v:
Biological Research, Vol 56, Iss 1, Pp 1-9 (2023)
Abstract Background The genomes of present-day non-Africans are composed of 1–3% of Neandertal-derived DNA as a consequence of admixture events between Neandertals and anatomically modern humans about 50–60 thousand years ago. Neandertal-introgre
Externí odkaz:
https://doaj.org/article/5ff8a3d541ef46499f490cb19c62c412
Autor:
Rima Nabbout, Galliano Zanello, Dixie Baker, Lora Black, Isabella Brambilla, Orion J. Buske, Laurie S. Conklin, Elin Haf Davies, Daria Julkowska, Yeonju Kim, Thomas Klopstock, Harumasa Nakamura, Kim G. Nielsen, Anne R. Pariser, Jose Carlos Pastor, Maurizio Scarpa, Maureen Smith, Domenica Taruscio, Stephen Groft
Publikováno v:
Orphanet Journal of Rare Diseases, Vol 18, Iss 1, Pp 1-10 (2023)
Abstract Background Many patients with rare diseases are still lacking a timely diagnosis and approved therapies for their condition despite the tremendous efforts of the research community, biopharmaceutical, medical device industries, and patient s
Externí odkaz:
https://doaj.org/article/c9ca331354bc45bb934c597b9e06fa26
Autor:
Giulia Stazi, Ludovica Taglieri, Alice Nicolai, Annalisa Romanelli, Rossella Fioravanti, Stefania Morrone, Manuela Sabatino, Rino Ragno, Samanta Taurone, Marcella Nebbioso, Raffaella Carletti, Marco Artico, Sergio Valente, Susanna Scarpa, Antonello Mai
Publikováno v:
Clinical Epigenetics, Vol 15, Iss 1, Pp 1-2 (2023)
Externí odkaz:
https://doaj.org/article/893e76ca84e042c89d1c43513000141b
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Autor:
Brocco Giorgio, Bonora Antonio, Barbi Stefano, Furlan Federico, Mafficini Andrea, Sorio Claudio, Blasi Francesco, Talamini Giorgio, Bassi Claudio, Scarpa Aldo
Publikováno v:
BMC Cancer, Vol 11, Iss 1, p 448 (2011)
Abstract Background The urokinase plasminogen activator receptor is highly expressed and its gene is amplified in about 50% of pancreatic ductal adenocarcinomas; this last feature is associated with worse prognosis. It is unknown whether the level of
Externí odkaz:
https://doaj.org/article/7877a64ead924d568224ad546067ede5