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 recommendations on cerliponase alfa (Brineura) for treating neuronal ceroid lipofuscinosis type 2 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 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
In development [GID-IPG10145] Expected publication date: 05 May 2021
In development [GID-IPG10205] Expected publication date: TBC
In development [GID-IPG10203] Expected publication date: TBC
In development [GID-IPG10204] Expected publication date: TBC
In development [GID-IP1131] Expected publication date: TBC
In development [GID-IP1180] Expected publication date: TBC
In development [GID-IPG10158] Expected publication date: TBC
In development [GID-IPG10160] Expected publication date: TBC
In development [GID-IPG10163] Expected publication date: TBC
In development [GID-IPG10166] Expected publication date: TBC
In development [GID-IPG10181] Expected publication date: TBC
In development [GID-IPG10179] Expected publication date: TBC
In development [GID-IPG10180] Expected publication date: TBC
In development [GID-IPG10191] Expected publication date: TBC
In development [GID-IPG10192] Expected publication date: TBC
In development [GID-IPG10193] Expected publication date: TBC
In development [GID-IPG10194] Expected publication date: TBC
In development [GID-IPG10195] Expected publication date: TBC
In development [GID-IPG10196] Expected publication date: TBC
In development [GID-IPG10197] Expected publication date: TBC
In development [GID-IPG10201] Expected publication date: TBC
In development [GID-IPG10202] Expected publication date: TBC
In development [GID-IPG10198] Expected publication date: TBC
In development [GID-IPG10199] Expected publication date: TBC
In development [GID-IPG10200] Expected publication date: TBC
In development [GID-IPG10188] Expected publication date: TBC
In development [GID-IPG10189] Expected publication date: TBC
In development [GID-IPG10190] Expected publication date: TBC
In development [GID-IP1046] Expected publication date: TBC
In development [GID-IPG10184] Expected publication date: TBC
In development [GID-IPG10187] Expected publication date: TBC
In development [GID-IPG10143] Expected publication date: 28 April 2021
In development [GID-IP1238] Expected publication date: TBC
In development [GID-IP2803] Expected publication date: TBC
In development [GID-IPG10168] Expected publication date: TBC
In development [GID-IPG10169] Expected publication date: TBC
In development [GID-IPG10170] Expected publication date: TBC
In development [GID-IPG10173] Expected publication date: TBC
In development [GID-IPG10174] Expected publication date: TBC