A Systematic Review and Meta-Analysis of Enzyme Replacement Therapy in Late-Onset Pompe Disease.

Alícia Dorneles DornellesAna Paula Pedroso JungesTiago Veiga PereiraBárbara Corrêa KrugCandice Beatriz Treter GonçalvesJuan Clinton LlerenaPriya Sunil KishnaniHaliton Alves de OliveiraIda Vanessa Doederlein Schwartz

Published in: Journal of clinical medicine (2021)

Considering the limitations imposed by the rarity of PD, our data suggest that GAA ERT improves 6MWT, physical QOL, and TOV in LOPD patients. ERT was safe in the studied population. PROSPERO register: 135102.

Keyphrases

late onset
replacement therapy
early onset
smoking cessation
ejection fraction
newly diagnosed
mental health
physical activity
prognostic factors
electronic health record
machine learning
patient reported outcomes
data analysis