North East and North Cumbria
ICS Formulary
ICS Formulary
9 Nutrition and blood
09-08-01 Pompe disease
| Avalglucosidase alfa Nexviadyme® |
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Formulary
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 NICE TA821: Avalglucosidase alfa for treating Pompe disease |
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| Cipaglucosidase alfa (CIPA) Pombiliti® |
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Formulary
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NICE TA912: Cipaglucosidase alfa with miglustat for treating late-onset Pompe disease |
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Links
- Highly specialised technologies guidance HST4: Migalastat for treating Fabry disease
NENC Guidelines for the recognition and management of non-IgE cow’s milk protein allergy (CMA) in infants- NHSE Specialised Commissioning Policy 2577: Subcutaneous copper histidinate injections for presymptomatic neonates with classical Menkes disease
- NICE HST19 - Elosulfase alfa for treating mucopolysaccharidosis type 4A
- NICE HST23: Asfotase alfa for treating paediatric-onset hypophosphatasia
- NICE HST29: Velmanase alfa for treating alpha-mannosidosis
- NICE HST30: Sebelipase alfa for treating Wolman disease
- NICE HST5 Eliglustat for treating type 1 Gaucher disease
- NICE TA821: Avalglucosidase alfa for treating Pompe disease
- NICE TA912: Cipaglucosidase alfa with miglustat for treating late-onset Pompe disease
- NICE TA915: Pegunigalsidase alfa for treating Fabry’s disease
- NICE TA961: Sebelipase alfa for treating lysosomal acid lipase deficiency that is not Wolman disease (terminated appraisal)