NICE HSTs
- 05/03/2020
- HST5: Eliglustat for treating type 1 Gaucher disease
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Published date: Next review: 2020
- 27/02/2020
- HST4: Migalastat for treating Fabry disease
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Evidence-based recommendations on migalastat (Galafold) for treating Fabry disease in people over 16.
- 27/02/2020
- HST2: Elosulfase alfa for treating mucopolysaccharidosis type IVa
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Evidence-based recommendations on elosulfase alfa (Vimizim) for treating mucopolysaccharidosis type IVa in adults and children.
- 27/02/2020
- HST1: Eculizumab for treating atypical haemolytic uraemic syndrome
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Evidence-based recommendations on eculizumab (Soliris) for treating atypical haemolytic uraemic syndrome in adults and children.
- 23/05/2019
- HST3: Ataluren for treating Duchenne muscular dystrophy with a nonsense mutation in the dystrophin gene
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Evidence-based recommendations on ataluren (Translarna) for treating Duchenne muscular dystrophy with a nonsense mutation in the dystrophin gene in people aged 5 years and older who can walk