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

Issue title: Abstracts from the 7th European Symposium on Steps Forward in Pompe Disease: Turin, Italy, November 21–22, 2014

Article type: Research Article

Authors: Chien, Yin-Hsiu | van der Ploeg, Ans | Jones, Simon | Byrne, Barry | Vellodi, Ashok | Leslie, Nancy | Mengel, Eugen | Shankar, Suma P. | Tanpaiboon, Pranoot | Stockton, David W. | Hennermann, Julia B. | Devecseri, Zsuzsanna | Kempf, Judy | Keutzer, Joan | Kishnani, Priya

Affiliations: National Taiwan University Hospital, Taipei, Taiwan | Department of Pediatrics & Center for Lysosomal and Metabolic Diseases, Erasmus MC University Medical Center, Rotterdam, Netherlands | Manchester Center for Genomic Medicine, CMFT, University of Manchester, UK | Department of Pediatrics, University of Florida, Gainesville, FL, USA | Great Ormond Street Hospital for Children NHS Foundation Trust, London, UK | Cincinnati Children's Hospital Medical Center, Cincinnati, OH, USA | University of Mainz, Mainz, Germany | Emory University School of Medicine, Atlanta, GA, USA | Children's National Medical Center, Washington, DC, USA | Wayne State University School of Medicine, Detroit, MI, USA | Charite Universitaetsmedizin Berlin, Berlin, Germany | Genzyme, a Sanofi company, Cambridge, MA, USA | Division of Medical Genetics, Department of Pediatrics, Duke University Medical Center, Durham, NC, USA

Note: [] Correspondence to: Yin-Hsiu Chien, National Taiwan University Hospital, Taipei, Taiwan. E-mail: [email protected]

DOI: 10.3233/JND-159053

Journal: Journal of Neuromuscular Diseases, vol. 2, no. s1, pp. S61-S62, 2015