Development of a novel tool for individual treatment trials in mucopolysaccharidosis.
Wiesinger AM, Bigger B, Giugliani R, Lampe C, Scarpa M, Moser T, Kampmann C, Zimmermann G, Lagler FB.
Wiesinger AM, et al.
J Inherit Metab Dis. 2024 Nov 21. doi: 10.1002/jimd.12816. Online ahead of print.
J Inherit Metab Dis. 2024.
PMID: 39572375
Mucopolysaccharidosis (MPS) encompasses a group of genetic lysosomal storage disorders, linked to reduced life expectancy and a significant lack of effective treatment options. ...The iterative methodology of our novel decision analysis framework (DAF) involves three key s …
Mucopolysaccharidosis (MPS) encompasses a group of genetic lysosomal storage disorders, linked to reduced life expectancy and a signi …