Evidence-based recommendations on culizumab (Soliris) for treating atypical haemolytic uraemic syndrome (aHUS)
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 (ADA–SCID)
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 migalastat (Galafold) for treating Fabry disease in people over 16
Evidence-based recommendations on ataluren (Translarna) for treating Duchenne muscular dystrophy with a nonsense mutation in the dystrophin gene..
Evidence-based recommendations on elosulfase alfa (Vimizim) for treating mucopolysaccharidosis type IVa (MPS IVA)
Evidence-based recommendations on voretigene neparvovec (Luxturna) for RPE65-mediated inherited retinal dystrophies (confirmed biallelic RPE65 gene mutations)
Evidence-based recommendations on patisiran (Onpattro) for treating hereditary transthyretin amyloidosis in adults with stage 1 and stage 2 polyneuropathy
Evidence-based recommendations on cerliponase alfa (Brineura) for treating neuronal ceroid lipofuscinosis type 2 (CLN2) in children
Proposed [GID-HST10020] Expected publication date: TBC
In development [GID-HST10010] Expected publication date: TBC
In development [GID-HST10027] Expected publication date: 11 November 2020
Proposed [GID-HST10022] Expected publication date: TBC
Proposed [GID-HST10023] Expected publication date: TBC
Proposed [GID-HST10019] Expected publication date: TBC
In development [GID-HST10009] Expected publication date: TBC
In development [GID-LYSOSOMALACIDLIPASEDEFICIENCYSEBELIPASEALFAID737] Expected publication date: TBC
In development [GID-HST10017] Expected publication date: TBC
In development [GID-HST10015] Expected publication date: 03 June 2020
In development [GID-HST10011] Expected publication date: 13 January 2021
In development [GID-HST10026] Expected publication date: 08 September 2020
We are listening to your views on this highly specialised technology. Comments close 27 January 2020.
Proposed [GID-HST10028] Expected publication date: TBC
In development [GID-HST10004] Expected publication date: TBC
In development [GID-HST10007] Expected publication date: TBC