Metreleptin for treating lipodystrophy (HST14)
Evidence-based recommendations on metreleptin (Myalepta) for treating lipodystrophy in children and adults
Search results
14 results
-
-
Volanesorsen for treating familial chylomicronaemia syndrome (HST13)
Evidence-based recommendations on volanesorsen (Waylivra) for treating familial chylomicronaemia syndrome in adults
-
Cerliponase alfa for treating neuronal ceroid lipofuscinosis type 2 (HST12)
Evidence-based recommendations on cerliponase alfa (Brineura) for treating neuronal ceroid lipofuscinosis type 2 in children
-
Voretigene neparvovec for treating inherited retinal dystrophies caused by RPE65 gene mutations (HST11)
Evidence-based recommendations on voretigene neparvovec (Luxturna) for RPE65-mediated inherited retinal dystrophies in people with vision loss caused by inherit
-
Patisiran for treating hereditary transthyretin amyloidosis (HST10)
Evidence-based recommendations on patisiran (Onpattro) for treating hereditary transthyretin amyloidosis in adults with stage 1 and stage 2 polyneuropathy
-
Inotersen for treating hereditary transthyretin amyloidosis (HST9)
Evidence-based recommendations on inotersen (Tegsedi) for stage 1 and stage 2 polyneuropathy in adults with hereditary transthyretin amyloidosis
-
Burosumab for treating X-linked hypophosphataemia in children and young people (HST8)
Evidence-based recommendations on burosumab (Crysvita) for treating X-linked hypophosphataemia (XLH) in children and young people
-
Strimvelis for treating adenosine deaminase deficiency–severe combined immunodeficiency (HST7)
Evidence-based recommendation on Strimvelis for adenosine deaminase deficiency–severe combined immunodeficiency
-
Asfotase alfa for treating paediatric-onset hypophosphatasia (HST6)
Evidence-based recommendations on asfotase alfa (Strensiq) for treating paediatric-onset hypophosphatasia in adults and children
-
Eliglustat for treating type 1 Gaucher disease (HST5)
Evidence-based recommendations on eliglustat (Cerdelga) for treating type 1 Gaucher disease in adults
-
Migalastat for treating Fabry disease (HST4)
Evidence-based recommendations on migalastat (Galafold) for treating Fabry disease in people over 16
-
Ataluren for treating Duchenne muscular dystrophy with a nonsense mutation in the dystrophin gene (HST3)
Evidence-based recommendations on ataluren (Translarna) for treating Duchenne muscular dystrophy with a nonsense mutation in the dystrophin gene in people aged
-
Elosulfase alfa for treating mucopolysaccharidosis type IVa (HST2)
Evidence-based recommendations on elosulfase alfa (Vimizim) for treating mucopolysaccharidosis type IVa in adults and children
-
Eculizumab for treating atypical haemolytic uraemic syndrome (HST1)
Evidence-based recommendations on eculizumab (Soliris) for treating atypical haemolytic uraemic syndrome in adults and children