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16 results for alpha-Glucosidases+-+adverse+effects

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Safety and efficacy of cipaglucosidase alfa plus miglustat versus alglucosidase alfa plus placebo in late-onset Pompe disease (PROPEL): an international, randomised, double-blind, parallel-group, phas...
Author / Creator:Schoser, Benedikt ; Roberts, Mark ; Byrne, Barry J ; Sitaraman, Sheela ; Jiang, Hai ; Laforêt, Pascal ; Toscano, Antonio ; Castelli, Jeff ; Díaz-Manera, Jordi ; Goldman, Mitchell ; van der Ploeg, Ans T ; Bratkovic, Drago ; Kuchipudi, Srilakshmi ; Mozaffar, Tahseen ; Kishnani, Priya S ; Sebok, Agnes ; Pestronk, Alan ; Dominovic-Kovacevic, Aleksandra ; Khan, Aneal ; Koritnik, Blaž ; Tard, Celine ; Lindberg, Christopher ; Quinn, Colin ; Eldridge, Crystal ; Bodkin, Cynthia ; Reyes-Leiva, David ; Hughes, Derralynn ; Stefanescu, Ela ; SALORT-CAMPANA, Emmanuelle ; Butler, Ernest ; Bouhour, Francoise ; Kim, Gee ; Konstantinos Papadimas, George ; Parenti, Giancarlo ; Bartosik-Psujek, Halina ; Kushlaf, Hani ; Akihiro, Hashiguchi ; Lau, Heather ; Pedro, Helio ; Andersen, Henning ; Amartino, Hernan ; Shiraishi, Hideaki ; Kobayashi, Hiroshi ; Tarnev, Ivaylo ; Vengoechea, Jaime ; Avelar, Jennifer ; Shin, Jin-Hong ; Cauci, Jonathan ; Alonso-Pérez, Jorge ; Janszky, Jozsef ; Berthy, Julie ; Cornelia, Kornblum ; Gutschmidt, Kristina ; Claeys, Kristl ; Judit Molnar, Maria ; Wencel, Marie ; Tarnopolsky, Mark ; Dimachkie, Mazen ; Tchan, Michel ; Freimer, Miriam ; Longo, Nicola ; Vidal-Fernandez, Nuria ; Musumeci, Olimpia ; Goker-Alpan, Ozlem ; Deegan, Patrick ; Clemens, Paula R. ; Roxburgh, Richard ; Henderson, Robert ; Hopkin, Robert ; Sacconi, Sabrina ; Fecarotta, Simona ; Attarian, Shahram ; Wenninger, Stephan ; Dearmey, Stephanie ; Hiwot, Tarekegn ; Burrow, Thomas ; Ruck, Tobias ; Sawada, Tomo ; Laszlo, Vescei ; Löscher, Wolfgang ; Chien, Yin-Hsiu
Publisher:England: Elsevier Ltd
Journal title:Lancet neurology, 2021-12, Vol.20 (12), p.1027-1037
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A Randomized Study of Alglucosidase Alfa in Late-Onset Pompe's Disease
Author / Creator:van der Ploeg, Ans T ; Clemens, Paula R ; Corzo, Deyanira ; Escolar, Diana M ; Florence, Julaine ; Groeneveld, Geert Jan ; Herson, Serge ; Kishnani, Priya S ; Laforet, Pascal ; Lake, Stephen L ; Lange, Dale J ; Leshner, Robert T ; Mayhew, Jill E ; Morgan, Claire ; Nozaki, Kenkichi ; Park, Dorothy J ; Pestronk, Alan ; Rosenbloom, Barry ; Skrinar, Alison ; van Capelle, Carine I ; van der Beek, Nadine A ; Wasserstein, Melissa ; Zivkovic, Sasa A
Publisher:Waltham, MA: Massachusetts Medical Society
Journal title:The New England journal of medicine, 2010-04, Vol.362 (15), p.1396-1406
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104-week efficacy and safety of cipaglucosidase alfa plus miglustat in adults with late-onset Pompe disease: a phase III open-label extension study (ATB200-07)
Author / Creator:Schoser, Benedikt ; Kishnani, Priya S. ; Bratkovic, Drago ; Byrne, Barry J. ; Claeys, Kristl G. ; Díaz-Manera, Jordi ; Laforêt, Pascal ; Roberts, Mark ; Toscano, Antonio ; van der Ploeg, Ans T. ; Castelli, Jeff ; Goldman, Mitchell ; Holdbrook, Fred ; Sitaraman Das, Sheela ; Wasfi, Yasmine ; Mozaffar, Tahseen
Publisher:Berlin/Heidelberg: Springer Berlin Heidelberg
Journal title:Journal of neurology, 2024-05, Vol.271 (5), p.2810-2823
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Enzyme replacement therapy with alglucosidase alfa in 44 patients with late-onset glycogen storage disease type 2: 12-month results of an observational clinical trial
Author / Creator:Strothotte, S. ; Strigl-Pill, N. ; Grunert, B. ; Kornblum, C. ; Eger, K. ; Wessig, C. ; Deschauer, M. ; Breunig, F. ; Glocker, F. X. ; Vielhaber, S. ; Brejova, A. ; Hilz, M. ; Reiners, K. ; Müller-Felber, W. ; Mengel, E. ; Spranger, M. ; Schoser, Benedikt
Publisher:Heidelberg: D. Steinkopff-Verlag
Journal title:Journal of neurology, 2010-01, Vol.257 (1), p.91-97
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Pompe disease in adulthood: effects of antibody formation on enzyme replacement therapy
Author / Creator:de Vries, Juna M. ; Kuperus, Esther ; Hoogeveen-Westerveld, Marianne ; Kroos, Marian A. ; Wens, Stephan C.A. ; Stok, Merel ; van der Beek, Nadine A.M.E. ; Kruijshaar, Michelle E. ; Rizopoulos, Dimitris ; van Doorn, Pieter A. ; van der Ploeg, Ans T. ; Pijnappel, W.W.M. Pim
Publisher:New York: Elsevier Inc
Journal title:Genetics in medicine, 2017-01, Vol.19 (1), p.90-97
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Population pharmacokinetic modeling and dosing simulation of avalglucosidase alfa for selecting alternative dosing regimen in pediatric patients with late-onset pompe disease
Author / Creator:Tiraboschi, Gilles ; Marchionni, David ; Tuffal, Gilles ; Fabre, David ; Martinez, Jean-Marie ; Haack, Kristina An ; Miossec, Patrick ; Kittner, Barbara ; Daba, Nadia ; Hurbin, Fabrice
Publisher:New York: Springer US
Journal title:Journal of pharmacokinetics and pharmacodynamics, 2023-12, Vol.50 (6), p.461-474
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Efficacy, safety profile, and immunogenicity of alglucosidase alfa produced at the 4,000-liter scale in US children and adolescents with Pompe disease: ADVANCE, a phase IV, open-label, prospective stu...
Author / Creator:Hahn, Si Houn ; Kronn, David ; Leslie, Nancy D ; Pena, Loren D M ; Tanpaiboon, Pranoot ; Gambello, Michael J ; Gibson, James B ; Hillman, Richard ; Stockton, David W ; Day, John W ; Wang, Raymond Y ; An Haack, Kristina ; Shafi, Raheel ; Sparks, Susan ; Zhao, Yang ; Wilson, Catherine ; Kishnani, Priya S
Publisher:New York: Nature Publishing Group US
Journal title:Genetics in medicine, 2018-10, Vol.20 (10), p.1284-1294
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A study on the safety and efficacy of reveglucosidase alfa in patients with late-onset Pompe disease
Author / Creator:Byrne, Barry J. ; Geberhiwot, Tarekegn ; Barshop, Bruce A. ; Barohn, Richard ; Hughes, Derralynn ; Bratkovic, Drago ; Desnuelle, Claude ; Laforet, Pascal ; Mengel, Eugen ; Roberts, Mark ; Haroldsen, Peter ; Reilley, Kristin ; Jayaram, Kala ; Yang, Ke ; Walsh, Liron
Publisher:England: BioMed Central Ltd
Journal title:Orphanet journal of rare diseases, 2017-08, Vol.12 (1), p.144-144, Article 144
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Immune responses to alglucosidase in infantile Pompe disease: recommendations from an Italian pediatric expert panel
Author / Creator:Gragnaniello, Vincenza ; Deodato, Federica ; Gasperini, Serena ; Donati, Maria Alice ; Canessa, Clementina ; Fecarotta, Simona ; Pascarella, Antonia ; Spadaro, Giuseppe ; Concolino, Daniela ; Burlina, Alberto ; Parenti, Giancarlo ; Strisciuglio, Pietro ; Fiumara, Agata ; Casa, Roberto Della
Publisher:England: BioMed Central
Journal title:Italian journal of pediatrics, 2022-03, Vol.48 (1), p.41-41, Article 41
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36 months observational clinical study of 38 adult Pompe disease patients under alglucosidase alfa enzyme replacement therapy
Author / Creator:Regnery, Caroline ; Kornblum, Cornelia ; Hanisch, Frank ; Vielhaber, Stefan ; Strigl-Pill, Nicola ; Grunert, Birgit ; Müller-Felber, Wolfgang ; Glocker, Franz Xaver ; Spranger, Matthias ; Deschauer, Marcus ; Mengel, Eugen ; Schoser, Benedikt
Publisher:Dordrecht: Springer Netherlands
Journal title:Journal of inherited metabolic disease, 2012-09, Vol.35 (5), p.837-845
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Long-term exposure to Myozyme results in a decrease of anti-drug antibodies in late-onset Pompe disease patients
Author / Creator:Masat, Elisa ; Laforêt, Pascal ; De Antonio, Marie ; Corre, Guillaume ; Perniconi, Barbara ; Taouagh, Nadjib ; Mariampillai, Kuberaka ; Amelin, Damien ; Mauhin, Wladimir ; Hogrel, Jean-Yves ; Caillaud, Catherine ; Ronzitti, Giuseppe ; Puzzo, Francesco ; Kuranda, Klaudia ; Colella, Pasqualina ; Mallone, Roberto ; Benveniste, Olivier ; Mingozzi, Federico ; Bassez, G. ; Bedat-Millet, A. L. ; Behin, A. ; Eymard, B. ; Leonard-Louis, S. ; Stojkovic, T. ; Canal, A. ; Decostre, V. ; Bouhour, F. ; Boyer, F. ; Castaing, Y. ; Chapon, F. ; Cintas, P. ; Durieu, I. ; Echaniz-Laguna, A. ; Feasson, L. ; Furby, A. ; Hamroun, D. ; Ferrer, X. ; Solé, G. ; Froissart, R. ; Piraud, M. ; Germain, D. ; Benistan, K. ; Guffon-Fouilhoux, N. ; Journel, H. ; Labauge, P. ; Lacour, A. ; Levy, A. ; Magot, A. ; Péréon, Y. ; Minot-Myhié, M. -C. ; Nadaj-Pakleza, A. ; Nathier, C. ; Orlikowski, D. ; Pellegrini, N. ; Petiot, P. ; Praline, J. ; Lofaso, F. ; Prigent, H. ; Dutry, A. ; Renard, D. ; Sacconi, S. ; Desnuelle, C. ; Salort-Campana, E. ; Pouget, J. ; Tiffreau, V. ; Vincent, D. ; Zagnoli, F.
Publisher:London: Nature Publishing Group UK
Journal title:Scientific reports, 2016-11, Vol.6 (1), p.36182-36182, Article 36182
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Desensitization of two young patients with infantile-onset Pompe disease and severe reactions to alglucosidase alfa
Author / Creator:Gragnaniello, V. ; Fecarotta, S. ; Pecoraro, A. ; Tarallo, A. ; Catzola, A. ; Spadaro, G. ; Parenti, G. ; Della Casa, R.
Publisher:Cham: Springer International Publishing
Journal title:Neurological sciences, 2019-07, Vol.40 (7), p.1453-1455
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Nephrotic Syndrome Complicating α-Glucosidase Replacement Therapy for Pompe Disease
Author / Creator:Hunley, Tracy E. ; Corzo, Deyanira ; Dudek, Martha ; Kishnani, Priya ; Amalfitano, Andrea ; Chen, Yuan-Tsong ; Richards, Susan M. ; Phillips, John A. ; Fogo, Agnes B. ; Tiller, George E.
Publisher:United States
Journal title:Pediatrics (Evanston), 2004-10, Vol.114 (4), p.e532-e535
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Enzyme therapy for Pompe disease with recombinant human α‐glucosidase from rabbit milk
Author / Creator:Van den Hout, J. M. P. ; Reuser, A. J. J. ; Klerk, J. B. C. ; Arts, W. F. ; Smeitink, J. A. M. ; Van der Ploeg, A. T.
Publisher:Dordrecht: Kluwer Academic Publishers
Journal title:Journal of inherited metabolic disease, 2001-04, Vol.24 (2), p.266-274
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24-Months results in two adults with Pompe disease on enzyme replacement therapy
Author / Creator:Vielhaber, Stefan ; Brejova, Andrea ; Debska-Vielhaber, Grazyna ; Kaufmann, Joern ; Feistner, Helmut ; Schoenfeld, Mircea A ; Awiszus, Friedemann
Publisher:Amsterdam: Elsevier B.V
Journal title:Clinical neurology and neurosurgery, 2011-06, Vol.113 (5), p.350-357
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First treatment for Pompe disease
Publisher:United States: U.S. Government Printing Office
Journal title:FDA consumer, 2006-07, Vol.40 (4), p.3-3
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Safety and efficacy of cipaglucosidase alfa plus miglustat versus alglucosidase alfa plus placebo in late-onset Pompe disease (PROPEL): an international, randomised, double-blind, parallel-group, phas...

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A Randomized Study of Alglucosidase Alfa in Late-Onset Pompe's Disease

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104-week efficacy and safety of cipaglucosidase alfa plus miglustat in adults with late-onset Pompe disease: a phase III open-label extension study (ATB200-07)

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Enzyme replacement therapy with alglucosidase alfa in 44 patients with late-onset glycogen storage disease type 2: 12-month results of an observational clinical trial

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Pompe disease in adulthood: effects of antibody formation on enzyme replacement therapy

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Population pharmacokinetic modeling and dosing simulation of avalglucosidase alfa for selecting alternative dosing regimen in pediatric patients with late-onset pompe disease

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Efficacy, safety profile, and immunogenicity of alglucosidase alfa produced at the 4,000-liter scale in US children and adolescents with Pompe disease: ADVANCE, a phase IV, open-label, prospective stu...

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A study on the safety and efficacy of reveglucosidase alfa in patients with late-onset Pompe disease

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Immune responses to alglucosidase in infantile Pompe disease: recommendations from an Italian pediatric expert panel

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36 months observational clinical study of 38 adult Pompe disease patients under alglucosidase alfa enzyme replacement therapy

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Long-term exposure to Myozyme results in a decrease of anti-drug antibodies in late-onset Pompe disease patients

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Desensitization of two young patients with infantile-onset Pompe disease and severe reactions to alglucosidase alfa

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Nephrotic Syndrome Complicating α-Glucosidase Replacement Therapy for Pompe Disease

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Enzyme therapy for Pompe disease with recombinant human α‐glucosidase from rabbit milk

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24-Months results in two adults with Pompe disease on enzyme replacement therapy

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First treatment for Pompe disease

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