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7 results for lysosomal
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Lysosomal acid lipase deficiency - sebelipase alfa [ID737]

In development [GID-LYSOSOMALACIDLIPASEDEFICIENCYSEBELIPASEALFAID737] Expected publication date: TBC

Highly specialised technologies guidance In development

Velmanase alfa for treating alpha-mannosidosis [ID800]

In development [GID-HST10010] Expected publication date: TBC

Highly specialised technologies guidance In development

Elosulfase alfa for treating mucopolysaccharidosis type IVa (HST2)

syndrome, and referred to in this document as MPS IVa) is an inherited lysosomal storage disease caused by a lack of the enzyme...

Technology appraisal guidance

Block scoping reports

block scoping report Batch 41 topic Sebelipase alfa for treating lysosomal acid lipase deficiency Batch 41 block scoping report

Published June 2018

Past appeals and decisions

TA124 Lung cancer (non-small cell) - pemetrexed 6 June 2007 TBC Lysosomal acid lipase deficiency - sebelipase alfa 25 April 2017 TA068...

Published November 2018

Migalastat for treating Fabry disease (HST4)

2 The condition 2.1 Fabry disease is an inherited lysosomal storage disease caused by a non-functional or only partially functional

Technology appraisal guidance

Eliglustat for treating type 1 Gaucher disease (HST5)

2 The condition 2.1 Gaucher disease is an inherited lysosomal storage disorder. It is caused by deficiency of the enzyme...

Technology appraisal guidance