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 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 onasemnogene abeparvovec (Zolgensma) for treating spinal muscular atrophy in babies
Evidence-based recommendations on givosiran (Givlaari) for treating acute hepatic porphyria in adults and young people aged 12 and over
Evidence-based recommendations on atidarsagene autotemcel (Libmeldy) for treating metachromatic leukodystrophy in children
Evidence-based recommendations on elosulfase alfa (Vimizim) for treating mucopolysaccharidosis type 4A in people of all ages
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
Evidence-based recommendations on odevixibat (Bylvay) for treating progressive familial intrahepatic cholestasis in people 6 months and older