Unwarranted, long term, alglucosidase alfa enzyme replacement therapy in two non-Pompe disease patients
- 25 June 2020
- journal article
- research article
- Published by Elsevier BV in Clinical Neurology and Neurosurgery
- Vol. 196, 106048
- https://doi.org/10.1016/j.clineuro.2020.106048
Abstract
No abstract availableKeywords
This publication has 4 references indexed in Scilit:
- Sensitivity of whole exome sequencing in detecting infantile- and late-onset Pompe diseaseMolecular Genetics and Metabolism, 2017
- Liquid Chromatography–Tandem Mass Spectrometry Assay of Leukocyte Acid α-Glucosidase for Post-Newborn Screening Evaluation of Pompe DiseaseClinical Chemistry, 2017
- Genetic heterozygosity and pseudodeficiency in the Pompe disease newborn screening pilot programMolecular Genetics and Metabolism, 2010
- A Randomized Study of Alglucosidase Alfa in Late-Onset Pompe's DiseaseThe New England Journal of Medicine, 2010