Survival and Developmental Milestones Among Pompe Registry Patients with Classic Infantile-Onset Pompe Disease with Different Timing of Initiation of Treatment with Enzyme Replacement Therapy

Affiliations

• ¹ National Taiwan University Hospital, Taipei, Taiwan.
• ² Department of Pediatrics & Center for Lysosomal and Metabolic Diseases, Erasmus MC University Medical Center, Rotterdam, Netherlands.
• ³ Manchester Center for Genomic Medicine, CMFT, University of Manchester, UK.
• ⁴ Department of Pediatrics, University of Florida, Gainesville, FL, USA.
• ⁵ Great Ormond Street Hospital for Children NHS Foundation Trust, London, UK.
• ⁶ Cincinnati Children's Hospital Medical Center, Cincinnati, OH, USA.
• ⁷ University of Mainz, Mainz, Germany.
• ⁸ Emory University School of Medicine, Atlanta, GA, USA.
• ⁹ Children's National Medical Center, Washington, DC, USA.
• ¹⁰ Wayne State University School of Medicine, Detroit, MI, USA.
• ¹¹ Charite Universitaetsmedizin Berlin, Berlin, Germany.
• ¹² Genzyme, a Sanofi company, Cambridge, MA, USA.
• ¹³ Division of Medical Genetics, Department of Pediatrics, Duke University Medical Center, Durham, NC, USA.