Givosiran for treating acute hepatic porphyria [ID1549]
In development [GID-HST10035] Expected publication date: TBC
In development [GID-HST10035] Expected publication date: TBC
In development [GID-HST10032] Expected publication date: TBC
In development [GID-HST10033] Expected publication date: TBC
In development [GID-HST10010] Expected publication date: TBC
In development [GID-HST10017] Expected publication date: TBC
In development [GID-HST10027] Expected publication date: TBC
In development [GID-LYSOSOMALACIDLIPASEDEFICIENCYSEBELIPASEALFAID737] Expected publication date: TBC
In development [GID-HST10009] Expected publication date: TBC
In development [GID-HST10028] Expected publication date: 13 August 2021
In development [GID-HST10026] Expected publication date: TBC
In development [GID-HST10043] Expected publication date: 22 January 2022
In development [GID-NG10113] Expected publication date: 19 January 2022
In development [GID-NG10145] Expected publication date: 27 April 2022
In development [GID-NG10156] Expected publication date: TBC
In development [GID-MT558] Expected publication date: 09 November 2021
In development [GID-MT561] Expected publication date: 02 February 2022
In development [GID-MT542] Expected publication date: 08 March 2021
In development [GID-MT526] Expected publication date: TBC
In development [GID-MT548] Expected publication date: 04 May 2021
In development [GID-MT539] Expected publication date: TBC
In development [GID-MT538] Expected publication date: TBC
In development [GID-MT544] Expected publication date: 15 September 2020
In development [GID-MT531] Expected publication date: TBC
In development [GID-MT551] Expected publication date: 05 October 2021
In development [GID-MT550] Expected publication date: 04 October 2021
In development [GID-MT553] Expected publication date: 30 November 2021
In development [GID-MT560] Expected publication date: 15 March 2022
In development [GID-MT556] Expected publication date: 23 February 2022
In development [GID-MT537] Expected publication date: 08 April 2021
In development [GID-MT562] Expected publication date: TBC
In development [GID-MT563] Expected publication date: TBC
In development [GID-MT554] Expected publication date: 06 January 2022
In development [GID-MT559] Expected publication date: 19 January 2022
In development [GID-MT557] Expected publication date: 22 March 2022
Evidence-based recommendations on eculizumab (Soliris) for treating atypical haemolytic uraemic syndrome in adults and children
Evidence-based recommendations on volanesorsen (Waylivra) for treating familial chylomicronaemia syndrome in adults
Evidence-based recommendations on elosulfase alfa (Vimizim) for treating mucopolysaccharidosis type IVa in adults and children
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 metreleptin (Myalepta) for treating lipodystrophy in children and adults
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 migalastat (Galafold) for treating Fabry disease in people over 16
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
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 recommendations on cerliponase alfa (Brineura) for treating neuronal ceroid lipofuscinosis type 2 in children
Evidence-based recommendations on Axonics sacral neuromodulation system for treating refractory overactive bladder
Evidence-based recommendations on SEM Scanner 200 for preventing pressure ulcers