Sfoglia per Autore
TCRαβ CD19 depletion in allogeneic haematopoietic stem cell transplantation performed for Hurler syndrome.
2016 Mainardi, Chiara; Tumino, M; Gazzola, Mv; Rampazzo, Angelica; Scarpa, Maurizio; Messina, Chiara
Targeted Polymeric Nanoparticles for Brain Delivery of High Molecular Weight Molecules in Lysosomal Storage Disorders
2016 Salvalaio, M; Rigon, Laura; Belletti, D; D'Avanzo, Francesca; Pederzoli, F; Ruozi, B; Marin, Oriano; Vandelli, Ma; Forni, F; Scarpa, Maurizio; Tomanin, Rosella; Tosi, G.
Glial degeneration with oxidative damage drives neuronal demise in MPSII disease
2016 Zalfa, Cristina; Verpelli, Chiara; D'Avanzo, Francesca; Tomanin, Rosella; Vicidomini, Cinzia; Cajola, Laura; Manara, Renzo; Sala, Carlo; Scarpa, Maurizio; Vescovi, Angelo Luigi; De Filippis, Lidia
Milde Mukopolysaccharidose-Formen
2016 Lampe, C; Scarpa, Maurizio; Bellettato, C. M.; Lampe, C.
Neuronopathic lysosomal storage disorders: Approaches to treat the central nervous system
2015 Scarpa, Maurizio; Bellettato, Cinzia Maria; Lampe, Christina; Begley, David J.
A Phase 3 Trial of Sebelipase Alfa in Lysosomal Acid Lipase Deficiency
2015 Burton, Barbara K; Balwani, Manisha; Feillet, François; Barić, Ivo; Burrow, T. Andrew; Camarena Grande, Carmen; Coker, Mahmut; Consuelo Sánchez, Alejandra; Deegan, Patrick; Di Rocco, Maja; Enns, Gregory M; Erbe, Richard; Ezgu, Fatih; Ficicioglu, Can; Furuya, Katryn N; Kane, John; Laukaitis, Christina; Mengel, Eugen; Neilan, Edward G; Nightingale, Scott; Peters, Heidi; Scarpa, Maurizio; Schwab, K. Otfried; Smolka, Vratislav; Valayannopoulos, Vassili; Wood, Marnie; Goodman, Zachary; Yang, Yijun; Eckert, Stephen; Rojas Caro, Sandra; Quinn, Anthony G.
Prevalence of Anti-Adeno-Associated Virus Serotype 8 Neutralizing Antibodies and Arylsulfatase B Cross-Reactive Immunologic Material in Mucopolysaccharidosis VI Patient Candidates for a Gene Therapy Trial
2015 Ferla, Rita; Claudiani, Pamela; Savarese, Marco; Kozarsky, Karen; Parini, Rossella; Scarpa, Maurizio; Donati, Maria Alice; Sorge, Giovanni; Hopwood, John J.; Parenti, Giancarlo; Fecarotta, Simona; Nigro, Vincenzo; Sivri, Hatice Serap; Van Der Ploeg, Ans; Andria, Generoso; Brunetti Pierri, Nicola; Auricchio, Alberto
Review and evaluation of the methodological quality of the existing guidelines and recommendations for inherited neurometabolic disorders Inherited metabolic diseases
2015 Cassis, Linda; Cortès Saladelafont, Elisenda; Molero Luis, Marta; Yubero, Delia; González, Maria Julieta; Herrero, Aida Ormazabal; Fons, Carme; Jou, Cristina; Sierra, Cristina; Castejon Ponce, Esperanza; Ramos, Federico; Armstrong, Judith; O'Callaghan, M. Mar; Casado, Mercedes; Montero, Raquel; Olivas, Silvia Maria Meavilla; Artuch, Rafael; Barić, Ivo; Bartoloni, Franco; Bellettato, Cinzia Maria; Bonifazi, Fedele; Ceci, Adriana; Cvitanović Šojat, Ljerka; Dali, Christine I; D'Avanzo, Francesca; Fumic, Ksenija; Giannuzzi, Viviana; Lampe, Christina; Scarpa, Maurizio; Garcia Cazorla, Ángels
Cystic fibrosis carrier screening effects on birth prevalence and newborn screening
2015 Castellani, Carlo; Picci, Luigi; Tridello, Gloria; Casati, Elia; Tamanini, Anna; Bartoloni, Lucia; Scarpa, Maurizio; Assael, Baroukh M.
Optimizing the molecular diagnosis of GALNS: Novel methods to define and characterize morquio-A syndrome-associated mutations
2015 Caciotti, Anna; Tonin, Rodolfo; Rigoldi, Miriam; Ferri, Lorenzo; Catarzi, Serena; Cavicchi, Catia; Procopio, Elena; Donati, Maria Alice; Ficcadenti, Anna; Fiumara, Agata; Barone, Rita; Garavelli, Livia; Rocco, Maja Di; Filocamo, Mirella; Antuzzi, Daniela; Scarpa, Maurizio; Mooney, Sean D.; Li, Biao; Skouma, Anastasia; Bianca, Sebastiano; Concolino, Daniela; Casalone, Rosario; Monti, Elena; Pantaleo, Marilena; Giglio, Sabrina; Guerrini, Renzo; Parini, Rossella; Morrone, Amelia
Clinical efficacy of Enzyme Replacement Therapy in paediatric Hunter patients, an independent study of 3.5 years
2014 Tomanin, Rosella; Zanetti, Alessandra; D'Avanzo, Francesca; Rampazzo, Angelica; Gasparotto, Nicoletta; Rossella, Parini; Antonia, Pascarella; Daniela, Concolino; Elena, Procopio; Agata, Fiumara; Borgo, Andrea; Frigo, ANNA CHIARA; Scarpa, Maurizio
A column-switching HPLC-MS/MS method for mucopolysaccharidosis type I analysis in a multiplex assay for the simultaneous newborn screening of six lysosomal storage disorders.
2014 Gucciardi, Antonina; Legnini, Elisa; Di Gangi, Im; Corbetta, C; Tomanin, Rosella; Scarpa, Maurizio; Giordano, Giuseppe
BBB Crossing in Lysosomal Storage Disorders: A Nanoparticle-Based Approach
2014 Rigon, Laura; Salvalaio, Marika; Tosi, Giovanni; Belletti, Daniela; D'Avanzo, Francesca; Ruozi, Barbara; Vandelli, Maria Angela; Forni, Flavio; Scarpa, Maurizio; Tomanin, Rosella
Intrathecal delivery of protein therapeutics to the brain: a critical reassessment
2014 Calias, Pericles; Banks, William A; Begley, David; Scarpa, Maurizio; Dickson, Patricia
A Hunter Patient with a Severe Phenotype Reveals Two Large Deletions and Two Duplications Extending 1.2 Mb Distally to IDS Locus
2014 Zanetti, Alessandra; Tomanin, Rosella; Rampazzo, Angelica; Rigon, Chiara; Gasparotto, Nicoletta; Cassina, Matteo; Clementi, Maurizio; Scarpa, Maurizio
Development of a Scoring System to Evaluate the Severity of Craniocervical Spinal Cord Compression in Patients with Mucopolysaccharidosis IVA (Morquio A Syndrome).
2013 Möllmann, C; Lampe, Cg; Müller Forell, W; Scarpa, Maurizio; Harmatz, P; Schwarz, M; Beck, M; Lampe, C.
Mucopolysaccharidoses and other lysosomal storage diseases.
2013 Lampe, C; Bellettato, Cm; Karabul, N; Scarpa, Maurizio
Circadian transcriptome analysis in human fibroblasts from Hunter syndrome and impact of iduronate-2-sulfatase treatment
2013 Mazzoccoli, G; Tomanin, Rosella; Mazza, T; D'Avanzo, Francesca; Salvalaio, Marika; Rigon, Laura; Zanetti, Alessandra; Pazienza, V; Francavilla, M; Giuliani, F; Vinciguerra, M; Scarpa, Maurizio
Human amniotic fluid stem cells protect rat lungs exposed to moderate hyperoxia.
2013 Grisafi, Davide; Pozzobon, Michela; Dedja, Arben; Vanzo, V; Tomanin, R; Porzionato, Andrea; Macchi, Veronica; Salmaso, Roberto; Scarpa, Maurizio; Cozzi, E; Fassina, Ambrogio; Navaglia, F; Maran, C; Onisto, Maurizio; Caenazzo, Luciana; DE COPPI, Paolo; DE CARO, Raffaele; Chiandetti, Lino; Zaramella, P.
Enzyme replacement therapy for mucopolysaccharidosis VI: long-term cardiac effects of galsulfase (Naglazyme®) therapy.
2013 Braunlin, E; Rosenfeld, H; Kampmann, C; Johnson, J; Beck, M; Giugliani, R; Guffon, N; Ketteridge, D; Sá Miranda, Cm; Scarpa, Maurizio; Schwartz, Iv; Leão Teles, E; Wraith, Je; Barrios, P; Dias da Silva, E; Kurio, G; Richardson, M; Gildengorin, G; Hopwood, Jj; Imperiale, M; Schatz, A; Decker, C; Harmatz, P; MPS VI Study, Group
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