Open AccessThe effectiveness and cost-effectiveness of enzyme and substrate replacement therapies: a longitudinal cohort study of people with lysosomal storage disorders.
Author(s) -
Katrina Wyatt,
William Henley,
Lindsey Anderson,
Rob Anderson,
Vasilis Nikolaou,
Kerstin Stein,
L Klinger,
D.A. Hughes,
S. Waldek,
Robin Lachmann,
Atul Mehta,
Ashok Vellodi,
Stuart Logan
Publication year - 2012
Publication title -
hta on dvd/health technology assessment
Language(s) - English
Resource type - Journals
SCImago Journal Rank - 1.426
H-Index - 126
eISSN - 2046-4924
pISSN - 1366-5278
DOI - 10.3310/hta16390
Subject(s) - enzyme replacement therapy , medicine , fabry disease , cohort , pediatrics , renal function , lysosomal storage disease , mucopolysaccharidosis type ii , disease , cohort study
To determine natural history and estimate effectiveness and cost of enzyme replacement therapy (ERT) and substrate replacement therapy (SRT) for patients with Gaucher disease, Fabry disease, mucopolysaccharidosis type I (MPS I), mucopolysaccharidosis type II (MPS II), Pompe disease and Niemann-Pick type C (NPC) disease.