The effectiveness and cost-effectiveness of enzyme and substrate replacement therapies: a longitudinal cohort study of people with lysosomal storage disorders. | Zendy

Katrina Wyatt | Zendy; William Henley | Zendy; Lindsey Anderson | Zendy; Rob Anderson | Zendy; Vasilis Nikolaou | Zendy; Kerstin Stein | Zendy; L Klinger | Zendy; Derralynn Hughes | Zendy; S. Waldek | Zendy; Robin Lachmann | Zendy; Atul Mehta | Zendy; Ashok Vellodi | Zendy; Stuart Logan | Zendy

Open Access

The 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,

Derralynn Hughes,

S. Waldek,

Robin Lachmann,

Atul Mehta,

Ashok Vellodi,

Stuart Logan

Publication year - 2012

Publication title -

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 , mucopolysaccharidosis type ii , lysosomal storage disease , cohort study , disease

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.

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