Safety and efficacy of avalglucosidase alfa versus alglucosidase alfa in patients with late-onset Pompe disease (COMET): a phase 3, randomised, multicentre trial

Author:

Diaz-Manera Jordi,Kishnani Priya S,Kushlaf Hani,Ladha Shafeeq,Mozaffar Tahseen,Straub Volker,Toscano Antonio,van der Ploeg Ans T,Berger Kenneth I,Clemens Paula R,Chien Yin-Hsiu,Day John W,Illarioshkin Sergey,Roberts Mark,Attarian Shahram,Borges Joao Lindolfo,Bouhour Francoise,Choi Young Chul,Erdem-Ozdamar Sevim,Goker-Alpan Ozlem,Kostera-Pruszczyk Anna,Haack Kristina An,Hug Christopher,Huynh-Ba Olivier,Johnson Judith,Thibault Nathan,Zhou Tianyue,Dimachkie Mazen M,Schoser Benedikt,Behin Anthony,Boentert Matthias,Carvalho Gerson,Chahin Nizar,Charrow Joel,Deegan Patrick,Durmus Tekce Hacer,Duval Fanny,Genge Angela,Gutmann Ludwig,Henderson Robert D,Hennermann Julia B,Hiwot Tarekegn,Hughes Derralynn,Karaa Amel,Karam Chafic,Kautzky-Willer Alexandra,Komaki Hirofumi,Laforet Pascal,Longo Nicola,Malinova Vera,Maré Ricardo,Maxit Clarisa,Mengel Eugen,Moggio Maurizio Gualtiero,Molnár Mária Judit,Mongini Tiziana Enrica,Nadaj-Pakleza Aleksandra,Nascimento Osorio Andres,Noury Jean-Baptiste,Oliveira Acary Souza Bulle,Parman Yesim,Pena Loren,Remiche Gauthier,Sciacco Monica,Shieh Perry B,Smith Cheryl,Stulnig Thomas,Taithe Frederic,Tard Céline,Tarnopolsky Mark,Vorgerd Matthias,Whitley Chester,Young Peter,Alonso-Pérez Jorge,Altemus Patricia,Aubé-Nathier Anne-Catherine,Avelar Jennifer B,Bailey Carrie,Bekircan-Kurt Can Ebru,Billy Jenny,Boschi Silvia,Brown Kathryn E,Carrera Garcia Laura,Chase Lauren,Cirne Hamilton,Danjoux Loïc,Davion Jean-Baptiste,DeArmey Stephanie,Fedotova Ekaterina,Gandolfo Eve,Grosz Zoltan,Guellec Dewi,Guettsches Anne-Katrin,Guglieri Michela,Hatcher Erin,Helms Sina,Hufgard-Leitner Miriam,Klyushnikov Sergey A.,Langton Jacqui,Linková Lenka,Mavroudakis Nicolas,Mazurová Stella,Mori Madoka,Müller-Miny Louisa,Musumeci Olimpia,Nance Christopher S,Natera-de Benito Daniel,Neel Robert,Niizawa Gabriela A,Noll Lauren,Ortega Erik,Pasnoor Mamatha,Pautot Vivien,Potulska-Chromik Anna,Pugliese Alessia,Questienne Claire,Ramos Lopes Margarida,Reyes-Leiva David,Riedl Michaela,Rugiero Marcelo Francisco,Salort-Campana Emmanuelle,Sgobbi Souza Paulo Victor,Sole Guilhem,Solera Luca,Souto Lopes Suzara,Specht Sabine,Statland Jeffrey,Swenson Andrea,Tan Chong Yew,Tizon Sónia,van der Beek N A M E,van Kooten Harmke A.,Wencel Marie,Wenninger Stephan,Zagnoli Fabien

Funder

Sanofi Genzyme

Publisher

Elsevier BV

Subject

Clinical Neurology

Reference36 articles.

1. How to describe the clinical spectrum in Pompe disease?;Gungor;Am J Med Genet A,2013

2. Clinical manifestation and natural course of late-onset Pompe's disease in 54 Dutch patients;Hagemans;Brain,2005

3. The new era of Pompe disease: advances in the detection, understanding of the phenotypic spectrum, pathophysiology, and management;Kishnani;Am J Med Genet C,2012

4. Pompe disease: glycogen storage disease type II, acid α-glucosidase (acid maltase) deficiency;Reuser,2018

5. Pompe's disease;van der Ploeg;Lancet,2008

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