Evidence-based recommendations on ataluren (Translarna) for treating Duchenne muscular dystrophy with a nonsense mutation in the dystrophin gene in people aged
Evidence-based recommendations on odevixibat (Bylvay) for treating progressive familial intrahepatic cholestasis in people 6 months and older
Evidence-based recommendations on givosiran (Givlaari) for treating acute hepatic porphyria in adults and young people aged 12 and over
Evidence-based recommendations on volanesorsen (Waylivra) for treating familial chylomicronaemia syndrome in adults
Evidence-based recommendations on metreleptin (Myalepta) for treating lipodystrophy in children and adults
Evidence-based recommendations on eculizumab (Soliris) for treating atypical haemolytic uraemic syndrome in adults and children
Evidence-based recommendations on onasemnogene abeparvovec (Zolgensma) for treating spinal muscular atrophy in babies
Evidence-based recommendations on elosulfase alfa (Vimizim) for treating mucopolysaccharidosis type 4A in people of all ages
Evidence-based recommendations on selumetinib (Koselugo) for treating symptomatic and inoperable plexiform neurofibromas associated with type 1 neurofibromatosis in children aged 3 and over
Evidence-based recommendations on setmelanotide (Imcivree) for treating obesity caused by LEPR or POMC deficiency in people 6 years and over
Evidence-based recommendations on migalastat (Galafold) for treating Fabry disease in people over 16
Evidence-based recommendations on atidarsagene autotemcel (Libmeldy) for treating metachromatic leukodystrophy in children
Evidence-based recommendations on inotersen (Tegsedi) for stage 1 and stage 2 polyneuropathy in adults with hereditary transthyretin amyloidosis
Evidence-based recommendations on burosumab (Crysvita) for treating X-linked hypophosphataemia (XLH) in children and young people
Evidence-based recommendation on Strimvelis for adenosine deaminase deficiency–severe combined immunodeficiency
Evidence-based recommendations on asfotase alfa (Strensiq) for treating paediatric-onset hypophosphatasia in adults and children
Evidence-based recommendations on eliglustat (Cerdelga) for treating type 1 Gaucher disease in adults
Evidence-based recommendations on cerliponase alfa (Brineura) for treating neuronal ceroid lipofuscinosis type 2 in children
Evidence-based recommendations on voretigene neparvovec (Luxturna) for RPE65-mediated inherited retinal dystrophies in people with vision loss caused by inherit
Evidence-based recommendations on patisiran (Onpattro) for treating hereditary transthyretin amyloidosis in adults with stage 1 and stage 2 polyneuropathy
In development [GID-HST10009] Expected publication date: TBC
In development [GID-HST10033] Expected publication date: TBC
In development [GID-HST10047] Expected publication date: TBC
Awaiting development [GID-HST10046] Expected publication date: 15 December 2022
In development [GID-HST10044] Expected publication date: 25 January 2023
In development [GID-LYSOSOMALACIDLIPASEDEFICIENCYSEBELIPASEALFAID737] Expected publication date: TBC
In development [GID-HST10052] Expected publication date: 29 March 2023
In development [GID-HST10010] Expected publication date: TBC
We are listening to your views on this Highly specialised technology. Comments close 16 August 2022.
In development [GID-HST10053] Expected publication date: 25 May 2023
Awaiting development [GID-HST10032] Expected publication date: TBC
Awaiting development [GID-HST10039] Expected publication date: TBC
Awaiting development [GID-HST10020] Expected publication date: TBC
Awaiting development [GID-HST10022] Expected publication date: TBC
Awaiting development [GID-HST10049] Expected publication date: TBC
In development [GID-HST10017] Expected publication date: TBC
In development [GID-HST10007] Expected publication date: TBC
In development [GID-HST10037] Expected publication date: TBC
In development [GID-HST10004] Expected publication date: TBC
In development [GID-HST10050] Expected publication date: TBC
In development [GID-HST10019] Expected publication date: TBC
In development [GID-HST10038] Expected publication date: TBC
This guidance has been updated and replaced by NICE’s highly specialised technologies guidance on elosulfase alfa for treating mucopolysaccharidosis type 4A (HST19).
In development [GID-HST10040] Expected publication date: TBC