Higher dose alglucosidase alfa is associated with improved overall survival in infantile-onset Pompe disease (IOPD): data from the Pompe Registry.

Priya S KishnaniDavid KronnShugo SuwazonoAlexander BroomfieldJuan LlerenaZuhair Nasser Al-HassnanJulie L BatistaKathryn M WilsonMagali PeriquetNadia DabaAndreas HahnYin-Hsiu Chien

Published in: Orphanet journal of rare diseases (2023)

Higher ALGLU doses were associated with significantly improved overall and invasive ventilator-free survival in IOPD. Results were consistent across sensitivity analyses.

Keyphrases

free survival
replacement therapy
late onset
smoking cessation
early onset
electronic health record
acute respiratory distress syndrome
mechanical ventilation
machine learning
data analysis