Open AccessSurvival and Developmental Milestones Among Pompe Registry Patients with Classic Infantile-Onset Pompe Disease with Different Timing of Initiation of Treatment with Enzyme Replacement Therapy
Author(s) -
YinHsiu Chien,
Ans van der Ploeg,
Simon W. Jones,
Barry Byrne,
Ashok Vellodi,
Nancy Leslie,
Eugen Mengel,
Suma Shankar,
Pranoot Tanpaiboon,
David W. Stockton,
Julia B. Hennermann,
Zsuzsanna Devecseri,
Judy Kempf,
Joan Keutzer,
Priya S. Kishnani
Publication year - 2015
Publication title -
journal of neuromuscular diseases
Language(s) - English
Resource type - Journals
SCImago Journal Rank - 1.366
H-Index - 23
eISSN - 2214-3602
pISSN - 2214-3599
DOI - 10.3233/jnd-159053
Subject(s) - enzyme replacement therapy , medicine , pediatrics , disease , developmental milestone , age of onset