Considerations for evaluating the effectiveness and long-term outcome of enzyme replacement therapy in Pompe disease

scientific article published on 07 August 2019

Considerations for evaluating the effectiveness and long-term outcome of enzyme replacement therapy in Pompe disease is …
instance of (P31):
editorialQ871232
scholarly articleQ13442814

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P356DOI10.3345/KJP.2018.07402
P932PMC publication ID7027348
P698PubMed publication ID31401827

P50authorChong Kun CheonQ47703410
P2860cites workClinical and molecular characterization of Korean children with infantile and late-onset Pompe disease: 10 years of experience with enzyme replacement therapy at a single centerQ57798439
Juvenile and adult-onset acid maltase deficiency in France: genotype-phenotype correlationQ73172404
A qualitative systematic overview of the measurement properties of functional walk tests used in the cardiorespiratory domainQ34131061
ATS StatementQ34136219
Clinical manifestation and natural course of late-onset Pompe's disease in 54 Dutch patientsQ34554310
Predicting cross-reactive immunological material (CRIM) status in Pompe disease using GAA mutations: lessons learned from 10 years of clinical laboratory testing experienceQ35752654
Pompe disease in infants and childrenQ35764507
An integrated approach to the interpretation of cardiopulmonary exercise testing.Q40704904
A retrospective, multinational, multicenter study on the natural history of infantile-onset Pompe diseaseQ44312356
Enzyme replacement therapy for infantile-onset Pompe disease.Q47385072
P275copyright licenseCreative Commons Attribution-NonCommercial 4.0 InternationalQ34179348
P6216copyright statuscopyrightedQ50423863
P433issue1
P304page(s)14-15
P577publication date2019-08-07
P1476titleConsiderations for evaluating the effectiveness and long-term outcome of enzyme replacement therapy in Pompe disease
P478volume63

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