P029: Early treatment with alglucosidase alfa is associated with improved survival in patients with infantile-onset Pompe disease: Data from Pompe Registry
Priya Kishnani,
David Stockton,
Andreas Hahn,
Juan Llerena,
Alexander Broomfield,
Julie Batista,
Meredith Foster,
Kathryn Wilson,
Susan Sparks,
Hannerieke van den Hout,
Yin-Hsiu Chien
Affiliations
Priya Kishnani
Division of Medical Genetics, Department of Pediatrics, Duke University Medical Center, Durham, NC
David Stockton
Division of Genetic, Genomic and Metabolic Disorders, Discipline of Pediatrics, Central Michigan University and Children's Hospital of Michigan, Detroit, MI
Andreas Hahn
Department of Child Neurology, University Hospital Giessen, Giessen, Germany
Juan Llerena
Centro de Genética Médica, Instituto Fernandes Figueira/FIOCRUZ, Rio de Janeiro, Brazil
Alexander Broomfield
Great Ormond Street Hospital, London, UK
Julie Batista
Sanofi, Cambridge, MA
Meredith Foster
Sanofi, Cambridge, MA
Kathryn Wilson
Sanofi, Cambridge, MA
Susan Sparks
Sanofi, Cambridge, MA
Hannerieke van den Hout
Division of Metabolic Diseases and Genetics, Department of Pediatrics, Erasmus University Medical Center-Sophia, Rotterdam, The Netherlands
Yin-Hsiu Chien
Department of Medical Genetics and Pediatrics, National Taiwan University Hospital, Taipei, Taiwan
