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NICE Guidance
Conditions and diseases
Genetic conditions
Genetic conditions: general and other
6 new
and
0 updated
products since January 2017.
Highly specialised technologies guidance
Ataluren for treating Duchenne muscular dystrophy with a nonsense mutation in the dystrophin gene
(HST3)
July 2016
Migalastat for treating Fabry disease
(HST4)
February 2017
Diagnostics guidance
Molecular testing strategies for Lynch syndrome in people with colorectal cancer
(DG27)
February 2017
In development
Cerliponase alfa for treating neuronal ceroid lipofuscinosis type 2 [ID943]
(GID-HST10008)
June 2018
Highly specialised technologies guidance
Lysosomal acid lipase deficiency - sebelipase alfa [ID737]
(GID-LYSOSOMALACIDLIPASEDEFICIENCYSEBELIPASEALFAID737)
TBC
Highly specialised technologies guidance
Strimvelis for treating severe combined immunodeficiency caused by adenosine deaminase deficiency [ID926]
(GID-HST10005)
February 2018
Highly specialised technologies guidance
Velmanase alfa for treating alpha-mannosidosis [ID800]
(GID-HST10010)
June 2018
Highly specialised technologies guidance