Evaluation of endocrinological involvement and metabolic status in patients with Gaucher disease Type 1 and Fabry disease under enzyme replacement therapy.
Melike ErsoyHamide PiskinpasaPublished in: Journal of pediatric endocrinology & metabolism : JPEM (2022)
Long time effective ERT seems to have beneficial effects on metabolic and hormonal status as well as primary target organs in both FD and GD1 patients.