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
Please note that any 'commercial in confidence' information has been removed.
If you have any questions about this page please email Michelle Adhemar - firstname.lastname@example.org
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