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)
LSDPEP – Medicines reviews recommendations
About this resource
Publication date:
Publication type:
Report
Language:
English