Listed below are the highly specialised technology evaluation block scoping reports which have been submitted to the Department of Health and Social Care.
These reports summarise the results of
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Batch 58 block scoping report
Batch 58 topic
- Inotersen for treating hereditary transthyretin-related amyloidosis.
- Patisiran for treating hereditary transthyretin-related amyloidosis.
- Volanesorsen for treating familial chylomicronaemia syndrome.
Batch 57 block scoping report
Batch 57 topic
- Human alpha1-proteinase inhibitor for treating emphysema.
Batch 56 block scoping report
Batch 56 topic
- Voretigene neparvovec for treating inherited retinal dystrophies caused by RPE65 gene mutations.
Batch 55 block scoping report
Batch 55 topic
- Burosumab for treating X-linked hypophosphataemia.
Batch 52 block scoping report
Batch 52 topics
- Metreleptin for treating lipodystrophy
- Afamelanotide for erythropoietic protoporphyria
Batch 51 block scoping report
Batch 51 topic
- Velmanase alfa for treating alpha-mannosidosis
Batch 50 block scoping report
Batch 50 topic
- Eteplirsen for treating Duchenne muscular dystrophy
- Cerliponase alfa for treating neuronal ceroid lipofuscinosis type 2
Batch 48 block scoping report
Batch 48 topic
- Alipogene tiparvovec for treating familial lipoprotein lipase deficiency
Batch 47 block scoping report
Batch 47 topic
- GSK2696273 for treating severe combined immunodeficiency caused by adenosine deaminase deficiency
Batch 44b block scoping report
Batch 44b topic
- Migalastat for treating Fabry disease
scoping report block
Batch 41 topic
- Sebelipase alfa for treating lysosomal acid lipase deficiency
Batch 40 block scoping report
Batch 40 topic
- Ataluren for treating Duchenne muscular dystrophy with a nonsense mutation in the dystrophin gene
Batch 37b block scoping report
Batch 37b topic
- Asfotase alfa for treating paediatric-onset hypophosphatasia
Batch 36 block scoping report
Batch 36 topic
- Elosulfase alfa for treating mucopolysaccharidosis type IVA
Batch 34 block scoping report
Batch 34 topic
- Eliglustat for treating type 1 Gaucher disease