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LSDPEP – Medicines reviews recommendations
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Publication date:
Date last updated:
Publication type:
Report
Language:
English
Description:
The independent review includes two 2 sections and eight 8 sub-sections of recommendations:
- Cross-program recommendations
- Medicine-specific recommendations
- Fabry disease
- Gaucher disease
- Mucopolysaccharidosis type I (MPS I)
- Mucopolysaccharidosis type II (MPS II)
- Mucopolysaccharidosis type VI (MPS VI)
- Pompe disease
- Hereditary tyrosinaemia type I (HT1)
- Paroxysmal nocturnal haemoglobinuria (PNH)