Sabine Specht
1 Papers
Sabine Specht is an academic researcher. The author has contributed to research in topics: Alglucosidase alfa & Population. The author has an hindex of 1, co-authored 1 publications.
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Papers
Safety and efficacy of avalglucosidase alfa versus alglucosidase alfa in patients with late-onset Pompe disease (COMET): a phase 3, randomised, multicentre trial.
Jordi Diaz-Manera,Priya S. Kishnani,Hani Kushlaf,Shafeeq Ladha,Tahseen Mozaffar,Volker Straub,Antonio Toscano,Ans T. van der Ploeg,Kenneth I. Berger,Paula R. Clemens,Paula R. Clemens,Yin-Hsiu Chien,John W. Day,Sergey Illarioshkin,Mark Roberts,Shahram Attarian,Joao Lindolfo Borges,Francoise Bouhour,Young Chul Choi,Sevim Erdem-Ozdamar,Ozlem Goker-Alpan,Anna Kostera-Pruszczyk,Kristina An Haack,Christopher Hug,Olivier Huynh-Ba,Judith Johnson,Nathan Thibault,Tianyue Zhou,Mazen M. Dimachkie,Benedikt Schoser,Anthony Behin,Matthias Boentert,Gerson Carvalho,Nizar Chahin,Joel Charrow,Patrick Deegan,Hacer Durmus Tekce,Fanny Duval,Angela Genge,Ludwig Gutmann,Robert D. Henderson,Julia B Hennermann,Tarekegn Hiwot,Derralynn Hughes,Amel Karaa,Chafic Karam,Alexandra Kautzky-Willer,Hirofumi Komaki,Pascal Laforet,Nicola Longo,Vera Malinova,Ricardo Maré,Clarisa Maxit,Eugen Mengel,Maurizio Gualtiero Moggio,Maria Judit Molnar,Tiziana Enrica Mongini,Aleksandra Nadaj-Pakleza,Andres Nascimento Osorio,Jean-Baptiste Noury,Acary Souza Bulle Oliveira,Yesim Parman,Loren D M Pena,Gauthier Remiche,Monica Sciacco,Perry B. Shieh,Cheryl Smith,Thomas Stulnig,Frederic Taithe,Céline Tard,Mark Tarnopolsky,Matthias Vorgerd,Chester Whitley,Peter Young,Jorge Alonso-Pérez,Patricia Altemus,Anne-Catherine Aubé-Nathier,Jennifer B Avelar,Carrie Bailey,Can Ebru Bekircan-Kurt,Jenny Billy,Silvia Boschi,Kathryn E Brown,Laura Carrera Garcia,Lauren Chase,Hamilton Cirne,Loïc Danjoux,Jean-Baptiste Davion,Stephanie Dearmey,Ekaterina Fedotova,Eve Gandolfo,Zoltan Grosz,Dewi Guellec,Anne-Katrin Guettsches,Michela Guglieri,Erin Hatcher,Sina Helms,Miriam Hufgard-Leitner,Sergey A. Klyushnikov,Jacqui Langton,Lenka Linková,Nicolas Mavroudakis,Stella Mazurová,Madoka Mori,Louisa Müller-Miny,Olimpia Musumeci,Christopher Nance,Daniel Natera-de Benito,Robert Neel,Gabriela A Niizawa,Lauren Noll,Erik Ortega,Mamatha Pasnoor,Vivien Pautot,Anna Potulska-Chromik,Alessia Pugliese,Claire Questienne,Margarida Ramos Lopes,David Reyes-Leiva,Michaela Riedl,Marcelo Rugiero,Emmanuelle Salort-Campana,Paulo Victor Sgobbi Souza,Guilhem Sole,Luca Solera,Suzara Souto Lopes,Sabine Specht,Jeffrey Statland,Andrea Swenson,Chong Yew Tan,Sónia Tizon,N A M E van der Beek,Harmke A. van Kooten,Marie Wencel,Stephan Wenninger,Fabien Zagnoli +135 more
TL;DR: In this paper, the safety and efficacy of avalglucosidase alfa, a recombinant human GAA enzyme replacement therapy specifically designed for enhanced mannose-6-phosphate-receptor targeting and enzyme uptake aimed at increased glycogen clearance, in patients with late-onset Pompe disease was assessed.
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