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1
Survival and long-term outcomes in late-onset Pompe disease following alglucosidase alfa treatment: a systematic review and meta-analysis
Survival and long-term outcomes in late-onset Pompe disease following alglucosidase alfa treatment: a systematic review and meta-analysis
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Survival and long-term outcomes in late-onset Pompe disease following alglucosidase alfa treatment: a systematic review and meta-analysis

Schoser, Benedikt ; Stewart, Andrew ; Kanters, Steve ; Hamed, Alaa ; Jansen, Jeroen ; Chan, Keith ; Karamouzian, Mohammad ; Toscano, Antonio

Journal of Neurology, 2017-04, Vol.264 (4), p.621-630 [Periódico revisado por pares]

Berlin/Heidelberg: Springer Berlin Heidelberg

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2
European consensus for starting and stopping enzyme replacement therapy in adult patients with Pompe disease: a 10‐year experience
European consensus for starting and stopping enzyme replacement therapy in adult patients with Pompe disease: a 10‐year experience
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European consensus for starting and stopping enzyme replacement therapy in adult patients with Pompe disease: a 10‐year experience

Ploeg, A. T. ; Kruijshaar, M. E. ; Toscano, A. ; Laforêt, P. ; Angelini, C. ; Lachmann, R. H. ; Pascual Pascual, S. I. ; Roberts, M. ; Rösler, K. ; Stulnig, T. ; Doorn, P. A. ; Van den Bergh, P. Y. K. ; Vissing, J. ; Schoser, B. ; Bembi, Bruno ; Broomfield, Alexander ; Boentert, Matthias ; Desnuelle, Claude ; Findling, Oliver ; Hahn, Andreas ; Díaz‐Manera, Jordi ; Hundsberger, Thomas ; Kornblum, Cornelia ; Labarthé, Franҫois ; Laforet, Pascal ; Mengel, Karl‐Eugen ; Mongini, Tiziana ; Muller‐Felber, Wolfgang ; Parenti, Giancarlo ; Pijnappel, W. Pim ; Preisler, Nicolai ; Sacconi, Sabrina ; Talim, Beril ; Tardieu, Marine ; Beek, Nadine A.M.E ; Wenninger, Stephan

European journal of neurology, 2017-06, Vol.24 (6), p.768-e31 [Periódico revisado por pares]

England: John Wiley & Sons, Inc

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3
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, phase 3 trial
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, phase 3 trial
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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, phase 3 trial

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

Lancet neurology, 2021-12, Vol.20 (12), p.1027-1037 [Periódico revisado por pares]

England: Elsevier Ltd

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4
In Utero Enzyme-Replacement Therapy for Infantile-Onset Pompe’s Disease
In Utero Enzyme-Replacement Therapy for Infantile-Onset Pompe’s Disease
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In Utero Enzyme-Replacement Therapy for Infantile-Onset Pompe’s Disease

Cohen, Jennifer L. ; Chakraborty, Pranesh ; Fung-Kee-Fung, Karen ; Schwab, Marisa E. ; Bali, Deeksha ; Young, Sarah P. ; Gelb, Michael H. ; Khaledi, Hamid ; DiBattista, Alicia ; Smallshaw, Stacey ; Moretti, Felipe ; Wong, Derek ; Lacroix, Catherine ; El Demellawy, Dina ; Strickland, Kyle C. ; Lougheed, Jane ; Moon-Grady, Anita ; Lianoglou, Billie R. ; Harmatz, Paul ; Kishnani, Priya S. ; MacKenzie, Tippi C.

The New England journal of medicine, 2022-12, Vol.387 (23), p.2150-2158 [Periódico revisado por pares]

United States: Massachusetts Medical Society

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5
Long-term benefit of enzyme replacement therapy in Pompe disease: A 5-year prospective study
Long-term benefit of enzyme replacement therapy in Pompe disease: A 5-year prospective study
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Long-term benefit of enzyme replacement therapy in Pompe disease: A 5-year prospective study

Kuperus, Esther ; Kruijshaar, Michelle E ; Wens, Stephan C.A ; de Vries, Juna M ; Favejee, Marein M ; van der Meijden, Jan C ; Rizopoulos, Dimitris ; Brusse, Esther ; van Doorn, Pieter A ; van der Ploeg, Ans T ; van der Beek, Nadine A.M.E

Neurology, 2017-12, Vol.89 (23), p.2365-2373 [Periódico revisado por pares]

United States: American Academy of Neurology

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6
Large variation in effects during 10 years of enzyme therapy in adults with Pompe disease
Large variation in effects during 10 years of enzyme therapy in adults with Pompe disease
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Large variation in effects during 10 years of enzyme therapy in adults with Pompe disease

Harlaar, Laurike ; Hogrel, Jean-Yves ; Perniconi, Barbara ; Kruijshaar, Michelle E ; Rizopoulos, Dimitris ; Taouagh, Nadjib ; Canal, Aurélie ; Brusse, Esther ; van Doorn, Pieter A ; van der Ploeg, Ans T ; Laforêt, Pascal ; van der Beek, Nadine A M E

Neurology, 2019-11, Vol.93 (19), p.e1756-e1767 [Periódico revisado por pares]

United States: Lippincott Williams & Wilkins

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7
Design and Pharmacological Chaperone Effects of N‑(4′-Phenylbutyl)-DAB Derivatives Targeting the Lipophilic Pocket of Lysosomal Acid α‑Glucosidase
Design and Pharmacological Chaperone Effects of N‑(4′-Phenylbutyl)-DAB Derivatives Targeting the Lipophilic Pocket of Lysosomal Acid α‑Glucosidase
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Design and Pharmacological Chaperone Effects of N‑(4′-Phenylbutyl)-DAB Derivatives Targeting the Lipophilic Pocket of Lysosomal Acid α‑Glucosidase

Kato, Atsushi ; Nakagome, Izumi ; Kise, Maki ; Yoshimura, Kousuke ; Tanaka, Nobutada ; Nash, Robert J. ; Fleet, George W. J. ; Kobayashi, Yota ; Ikeda, Hayato ; Okada, Takuya ; Toyooka, Naoki

Journal of medicinal chemistry, 2023-07, Vol.66 (13), p.9023-9039 [Periódico revisado por pares]

United States: American Chemical Society

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8
Long-term Safety and Efficacy of Avalglucosidase Alfa in Patients With Late-Onset Pompe Disease
Long-term Safety and Efficacy of Avalglucosidase Alfa in Patients With Late-Onset Pompe Disease
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Long-term Safety and Efficacy of Avalglucosidase Alfa in Patients With Late-Onset Pompe Disease

Dimachkie, Mazen M ; Barohn, Richard J ; Byrne, Barry ; Goker-Alpan, Ozlem ; Kishnani, Priya S ; Ladha, Shafeeq ; Laforêt, Pascal ; Mengel, Karl Eugen ; Peña, Loren D M ; Sacconi, Sabrina ; Straub, Volker ; Trivedi, Jaya ; Van Damme, Philip ; van der Ploeg, Ans T ; Vissing, John ; Young, Peter ; Haack, Kristina An ; Foster, Meredith ; Gilbert, Jane M ; Miossec, Patrick ; Vitse, Olivier ; Zhou, Tianyue ; Schoser, Benedikt

Neurology, 2022-08, Vol.99 (5), p.e536-e548 [Periódico revisado por pares]

United States: American Academy of Neurology

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9
A Randomized Study of Alglucosidase Alfa in Late-Onset Pompe's Disease
A Randomized Study of Alglucosidase Alfa in Late-Onset Pompe's Disease
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A Randomized Study of Alglucosidase Alfa in Late-Onset Pompe's Disease

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

The New England journal of medicine, 2010-04, Vol.362 (15), p.1396-1406 [Periódico revisado por pares]

Waltham, MA: Massachusetts Medical Society

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10
The emerging phenotype of late-onset Pompe disease: A systematic literature review
The emerging phenotype of late-onset Pompe disease: A systematic literature review
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The emerging phenotype of late-onset Pompe disease: A systematic literature review

Chan, Justin ; Desai, Ankit K. ; Kazi, Zoheb B. ; Corey, Kaitlyn ; Austin, Stephanie ; Hobson-Webb, Lisa D. ; Case, Laura E. ; Jones, Harrison N. ; Kishnani, Priya S.

Molecular genetics and metabolism, 2017-03, Vol.120 (3), p.163-172 [Periódico revisado por pares]

United States: Elsevier Inc

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