Clinical and event-based outcomes of patients with mucopolysaccharidosis VI receiving enzyme replacement therapy in Turkey: a case series.

Aslı İnciİlyas OkurLeyla TümerGürsel BiberoğluMurat ÖktemFatih Süheyl Ezgü

Published in: Orphanet journal of rare diseases (2021)

This case series shows the high disease burden of the MPS VI population in Turkey and provides a unique insight into their clinical journey based on real-life clinical and event-based data collected before and after initiation of ERT.

Keyphrases

replacement therapy
smoking cessation
type diabetes
machine learning
adipose tissue
electronic health record
big data
insulin resistance