Results 91 to 100 of about 1,275 (171)

Profile of idursulfase for the treatment of Hunter syndrome

Research and Reports in Endocrine Disorders, 2015
Simona Sestito, Ferdinando Ceravolo, Michele Grisolia, Elisa Pascale, Licia Pensabene, Daniela Concolino Department of Pediatrics, University Magna Graecia of Catanzaro, Catanzaro, Italy Abstract: Mucopolysaccharidosis type II (MPS II; Hunter syndrome) is a rare X-linked lysosomal storage disorder caused by deficiency of the enzyme iduronate-2 ...
Sestito S, Ceravolo F, Grisolia M, Pascale E, Pensabene L, Concolino D   +5 more
openaire   +5 more sources

International consensus on (ICON) anaphylaxis [PDF]

, 2014
ICON: Anaphylaxis provides a unique perspective on the principal evidence-based anaphylaxis guidelines developed and published independently from 2010 through 2014 by four allergy/immunology organizations.
Alvarez-Twose, Antonella Muraro, Arga, Armstrong, Arroabarren, Aziz Sheikh, Barnett, Bauer, Belhocine, Ben-Shoshan, Bilo, Bonadonna, Boyce, Boyle, Brockow, Brouwers, Brown, Brown, Brown, Brown, Caffarelli, Camargo, Campbell, Campbell, Campbell, Cetinkaya, Chad, Chiang, Choo, Cox, Cummings, Dana V. Wallace, de Silva, Dhami, Duke, Edwards, Ellis, Enarson, F. Estelle R. Simons, Field, Fineman, Fleischer, Gibbison, Gibbs, Golden, Gonzalez-Perez, Grabenhenrich, Graham Roberts, Harduar-Morano, Heinzerling, Hoffer, Iribarren, Jang, Jones, Kalesnikoff, Kelleher, Khodoun, Kim, Kim, Lafuente, Landsman-Blumberg, Ledit R.F. Ardusso, Lee, Li, Lieberman, Liew, Liew, Liu, Lotvall, Ludman, Lynette P. Shek, M Beatrice Bilò, Margitta Worm, Mario Sanchez-Borges, Mertes, Minciullo, Motohiro Ebisawa, Muller, Muraro, Ng, Nicklas, Noimark, Nurmatov, Panesar, Park, Perel, Phil Lieberman, Raimer, Ralston, Renaudin, Ribeiro-Vaz, Richard F. Lockey, Rudders, Rudders, Rueff, Rueff, Sala-Cunill, Sampson, Sampson, Sanchez-Borges, Sanchez-Borges, Schneider, Sheikh, Sheikh, Shemesh, Shen, Sicherer, Sicherer, Simons, Simons, Simons, Simons, Simons, Simons, Simons, Simons, Simons, Sole, Stoevesandt, Strohmeier, Takahashi, Tanno, Techapornroong, Tejedor Alonso, Thong, Topal, Triggiani, Vadas, Vadas, Vale, Valent, Vereda, Victoria Cardona, Wolbing, Wood, Wood, Worm, Yehia M. El-Gamal   +137 more
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CNS penetration of intrathecal-lumbar idursulfase in the monkey, dog and mouse: implications for neurological outcomes of lysosomal storage disorder. [PDF]

PLoS ONE, 2012
A major challenge for the treatment of many central nervous system (CNS) disorders is the lack of convenient and effective methods for delivering biological agents to the brain.
Pericles Calias, Mikhail Papisov, Jing Pan, Nancy Savioli, Vasily Belov, Yan Huang, Jason Lotterhand, Mary Alessandrini, Nan Liu, Alan J Fischman, Jan L Powell, Michael W Heartlein   +11 more
doaj   +1 more source

Safety Evaluation of Chronic Intrathecal Administration of Idursulfase-IT in Cynomolgus Monkeys [PDF]

Toxicologic Pathology, 2011
Recombinant human idursulfase, an intravenous enzyme replacement therapy indicated for treatment of somatic symptoms of mucopolysaccharidosis II (Hunter syndrome), is anticipated to have minimal benefit for the cognitive impairment associated with the severe phenotype.
Brian R, Felice, Teresa L, Wright, Robert B, Boyd, Mark T, Butt, Richard W, Pfeifer, Jing, Pan, Juan A, Ruiz, Michael W, Heartlein, Pericles, Calias   +8 more
openaire   +2 more sources

The role of enzyme replacement therapy in severe Hunter syndrome—an expert panel consensus [PDF]

, 2011
Intravenous enzyme replacement therapy (ERT) with idursulfase for Hunter syndrome has not been demonstrated to and is not predicted to cross the blood–brain barrier. Nearly all published experience with ERT with idursulfase has therefore been in patients
A Milligan, A Vellodi, Barbara Burton, BK Burton, BK Burton, DF Moore, DJ Coman, E Galan-Gomez, E Glamuzina, E Vafiadaki, EF Neufeld, EJ McKinnis, G Bach, Gudrun Schulze Frenking, I Moreira da Silva, ID Young, ID Young, ID Young, ID Young, J Aerde van, J Muenzer, J Muenzer, J Muenzer, J Muenzer, JE Wraith, JE Wraith, Joseph Muenzer, K Isogai, Lorne Clarke, M Schlander, Maria Verónica Muñoz Rojas, Maurizio Scarpa, Michael Beck, N Guffon, Olaf Bodamer, P Harmatz, P Li, Paul Harmatz, PS Kishnani, R Froissart, R Katz, R Martin, Roberto Giugliani, S Bagewadi, S Yatziv, Simon Jones, SL Goldenfum, SL Staba, VK Prasad   +48 more
core   +4 more sources

Evaluation of the long-term treatment efects of intravenous idursulfase in patients with mucopolysaccharidosis II (MPS II) using statistical modeling : data from the Hunter Outcome Survey (HOS) [PDF]

, 2021
Background: Mucopolysaccharidosis II (MPS II; Hunter syndrome) is a rare, life-limiting lysosomal storage disease caused by defcient iduronate-2-sulfatase activity.
Botha, Jaco, Burton, Barbara K., Giugliani, Roberto, Harmatz, Paul, Kampmann, Christoph, Muenzer, Joseph   +5 more
core  

Mucopolysaccharidosis type II (Hunter syndrome): a clinical review and recommendations for treatment in the era of enzyme replacement therapy [PDF]

, 2007
Mucopolysaccharidosis type II (MPS II; Hunter syndrome) is a rare X-linked recessive disease caused by deficiency of the lysosomal enzyme iduronate-2-sulphatase, leading to progressive accumulation of glycosaminoglycans in nearly all cell types, tissues ...
A Cooper, A Friso, A Vellodi, A Yoskovitch, AK Vine, Allan Meldgaard Lund, AN Sapadin, Ans T. Van der Ploeg, AS Narita, B Elsner, BJ Poorthuis, C Hunter, C Moores, CT Sasaki, D Rigante, DM Thappa, E Sidransky, F Baehner, FS Haddad, Gunilla Malm, I Brama, ID Young, J Muenzer, J Muenzer, J Spranger, J. Edmond Wraith, JE Peck, JE Wraith, Jiri Zeman, K Tuschl, KM Timms, KM Timms, L Gaitini, Linda De Meirleir, M Beck, M Casal, MA Cleary, MA Simmons, Maurizio Scarpa, Michael Beck, ML Bondeson, MM Sleeper, N DiFerrante, N Shinomiya, Nathalie Guffon, Olaf A. Bodamer, SE Leighton, T Hishitani, VJ Parsons   +48 more
core   +4 more sources

Development of idursulfase therapy for mucopolysaccharidosis type II (Hunter syndrome): the past, the present and the future

Drug Design, Development and Therapy, 2017
David AH Whiteman,* Alan Kimura* Research & Development, Shire Human Genetic Therapies, Inc., Lexington, MA, USA *These authors contributed equally to this work Abstract: Mucopolysaccharidosis type II (MPS II; Hunter syndrome; OMIM 309900) is
Whiteman DAH, Kimura A
doaj  

Mucopolysaccharidosis I, II, and VI: Brief review and guidelines for treatment [PDF]

, 2010
Mucopolysaccharidoses (MPS) are rare genetic diseases caused by the deficiency of one of the lysosomal enzymes involved in the glycosaminoglycan (GAG) breakdown pathway.
Albano LM, Aldenhoven M, Ana Cecília Azevedo, Ana Maria Martins, Anawis MA, Anbu A, Andressa Federhen, Angelina Acosta, Ard JL, Auclair D, Azevedo AC, Baenher F, Barton NW, Bielicki J, Bjoraker KJ, Boelens JJ, Boelens JJ, Carmen Bonfim, Carolina Souza, Charles Marques Lourenço, Clarke LA, Clarke LA, Coelho JC, Cox-Brikman J, Dafne Horovitz, De Jong JG, Denise Norato, Denize Bonfim, Diane Marinho, Dilber E, Durval Palhares, Emerson Santana Santos, Erlane Ribeiro, Eugênia Valadares, Flavio F. Alcantara, Frossairt R, Fuller M, Fuller M, Fábio Guarany, Garcia AR, Gisele Rosone de Lucca, Giugliani R, Giugliani R, Harmatz P, Harmatz P, Harmatz P, Harmatz P, Harmatz P, Hein LK, Helena Pimentel, Herskhovitz E, Hirth A, Hite SH, Isabel Neves de Souza, Iwata S, Jordão Correa Neto, José Carlos Fraga, José Eduardo Goes, José Maria Cabral, José Simionato, Juan Llerena Jr., Kakkis ED, Kakkis ED, Kakkis ED, Karageorgos L, Kim Chong Ae, Kim KH, Krivit W, Lange MC, Laura Jardim, Leistner S, Li P, Liane Giuliani, Litjens T, Louise Lapagesse Pinto, Lowry RB, Luiz Carlos Santana da Silva, Mara L. Santos, Marcelo Kerstenetzky, Maria Angela Moreira, Maria Verônica Muñoz Rojas, Martin PL, Martin R, Matte U, Matte U, McGill JJ, McKninkkis EJR, Meikle PJ, Muenzer J, Muenzer J, Muenzer J, Muenzer J, Muenzer J, Muñoz-Rojas MV, Márcia Ribeiro, Nelson J, Nelson J, Neufeld EF, Nicole Ruas, Ochiai T, Osvaldo Artigalás, Oudit GY, Oudit GY, Pastores GM, Patricia Barrios, Paulo Aranda, Pereira VG, Petry MF, Petry MF, Poorthuis BJ, Prasad VK, Rachel Honjo, Randall DR, Raquel Boy, Roberto Giugliani, Rogoyski A, Ronaldo Costa, Sanjurjo-Crespo P, Schumacher RG, Schwartz IVD, Sifuentes M, Silvio Gilberto A. Avilla, Simone Fagondes, Soliman OII, Staba SL, Swiedler SJ, Taiane Vieira, Tan CT, Tolar J, Turner CT, Tuschl K, Vellodi A, Vijay S, Wegrzyn G, Weisstein JS, Wraith JE, Wraith JE, Wraith JE, Wraith JE, Wraith JE, Zareba G   +140 more
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The relationship between anti-idursulfase antibody status and safety and efficacy outcomes in attenuated mucopolysaccharidosis II patients aged 5years and older treated with intravenous idursulfase

Molecular Genetics and Metabolism, 2013
In the pivotal phase II/III trial of idursulfase administered intravenously to treat mucopolysaccharidosis II, approximately half of the patients developed antibodies to idursulfase. This post-hoc analysis of data from the phase II/III trial and extension study examined the relationship between antibody status and outcomes.
Barbier, A.J., Bielefeld, B., Whiteman, D.A.H., Natarajan, M., Pano, A., Amato, D.A.   +5 more
openaire   +4 more sources