A phase III, open-label clinical trial evaluating pegunigalsidase alfa administered every 4 weeks in adults with Fabry disease previously treated with other enzyme replacement therapies
Source
Journal of inherited metabolic disease - ISSN 0141-8955- (2024) p.
Unexplained splenomegaly as a diagnostic marker for a rare but severe disease with an innovative and highly effective new treatment option : a case report
Source
Molecular Genetics and Metabolism Reports - ISSN 2214-4269-41 (2024) p. 1-4
Left ventricular hypertrophy : do not forget Fabry disease. Diagnostic work-up and differential diagnosis
Source
Acta cardiologica - ISSN 0001-5385-79:6 (2024) p. 642-649
What mothers know about newborn bloodspot screening and the sources they use to acquire this knowledge : a pilot study in Flanders
Source
Children - ISSN 2227-9067-10:9 (2023) p. 1-9
A new multiplex analysis of glucosylsphingosine and globotriaosylsphingosine in dried blood spots by tandem mass spectrometry
Source
Molecular Genetics and Metabolism Reports - ISSN 2214-4269-37 (2023) p. 1-8