Odevixibat for treating progressive familial intrahepatic cholestasis (HST17)
Evidence-based recommendations on odevixibat (Bylvay) for treating progressive familial intrahepatic cholestasis in people 6 months and older
Evidence-based recommendations on odevixibat (Bylvay) for treating progressive familial intrahepatic cholestasis in people 6 months and older
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 selumetinib (Koselugo) for treating symptomatic and inoperable plexiform neurofibromas associated with type 1 neurofibromatosis in children aged 3 and over
Evidence-based recommendations on givosiran (Givlaari) for treating acute hepatic porphyria in adults and young people aged 12 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 eculizumab (Soliris) for treating atypical haemolytic uraemic syndrome in adults and children
Evidence-based recommendations on onasemnogene abeparvovec (Zolgensma) for treating spinal muscular atrophy in babies
Evidence-based recommendations on elosulfase alfa (Vimizim) for treating mucopolysaccharidosis type 4A in people of all ages
Evidence-based recommendations on atidarsagene autotemcel (Libmeldy) for treating metachromatic leukodystrophy 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 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 migalastat (Galafold) for treating Fabry disease in people over 16
Evidence-based recommendations on electroconvulsive therapy (ECT) for treating catatonia, prolonged or severe manic episodes or schizophrenia in adults
Evidence-based recommendations on fulvestrant (Faslodex) for untreated locally advanced or metastatic (secondary) oestrogen-receptor positive breast cancer in
Evidence-based recommendations on romosozumab (EVENITY) for severe osteoporosis in people after menopause who are at high risk of fracture
Evidence-based recommendations on ceritinib (Zykadia) for untreated anaplastic lymphoma kinase (ALK)-positive advanced non-small-cell lung cancer (NSCLC)
Evidence-based recommendations on ixekizumab (Taltz) for moderate to severe plaque psoriasis in adults
Evidence-based recommendations on obeticholic acid (Ocaliva) for treating primary biliary cholangitis (CBC / cirrhosis) in adults
NICE is unable to make a recommendation about the use in the NHS of afatinib for advanced squamous non-small-cell lung cancer after platinum-based chemotherapy
Evidence-based recommendations on Holoclar for treating limbal stem cell deficiency in adults after eye burns
Evidence-based recommendations on glycoprotein IIb/IIIa inhibitors(abciximab [ReoPro], eptifibatide [Integrilin], tirofiban [Aggrastat]) for acute coronary
NICE was unable to make a recommendation about the use in the NHS of elotuzumab for previously treated multiple myeloma because no evidence submission was
NICE was unable to make a recommendation about the use in the NHS of tenofovir alafenamide for treating chronic hepatitis B because no evidence submission was
NICE was unable to make a recommendation about the use in the NHS of bevacizumab for epidermal growth factor receptor mutation-positive non-small-cell lung
NICE is unable to make a recommendation about the use of ibrutinib with bendamustine and rituximab for relapsed or refractory chronic lymphocytic leukaemia
NICE is unable to make a recommendation about the use in the NHS of alectinib for anaplastic lymphoma kinase-positive advanced non-small-cell lung cancer
Evidence-based recommendations on inhaled corticosteroids (ICSs) for chronic asthma in children aged under 12
Evidence-based recommendations on etanercept (Enbrel) for treating severe psoriasis in adults
Evidence-based recommendations on darvadstrocel (Alofisel; stem-cell treatment) for complex perianal fistulas with mildly or non-active luminal Crohn’s disease
NICE is unable to make a recommendation on abatacept (Orencia) for treating psoriatic arthritis after DMARDs in adults because of no evidence submission from
Evidence-based recommendations on autologous chondrocyte implantation (ACI) using chondrosphere (Spherox) for symptomatic articular knee cartilage defects
Evidence-based recommendations on lesinurad (Zurampic) for treating chronic hyperuricaemia (too much uric acid/urate in blood) in adults with gout
Evidence-based recommendations on on regorafenib (Stivarga) for previously treated gastrointestinal stromal tumours in adults
Evidence-based recommendations on autologous chondrocyte implantation (ACI) in people with symptomatic articular cartilage defects of the knee
Evidence-based recommendations on risdiplam (Evrysdi) for 5q spinal muscular atrophy (SMA) in adults and children aged 2 months and over.
Evidence-based recommendations on fedratinib (Inrebic) for treating disease-related splenomegaly or symptoms in myelofibrosis in adults
Evidence-based recommendations on arsenic trioxide (Trisenox) for treating acute promyelocytic leukaemia in adults
Evidence-based recommendations on brentuximab vedotin (Adcetris) for treating CD30-positive Hodgkin lymphoma in adults
Evidence-based recommendations on on midostaurin (Rydapt) for untreated FLT3-mutation-positive acute myeloid leukaemia (AML) in adults
Evidence-based recommendations on tocilizumab (RoActemra) for treating giant cell arteritis in adults
Evidence-based recommendations on eribulin (Halaven) for treating locally advanced or metastatic (secondary) breast cancer in adults who have had only 1 course
Evidence-based recommendations on ixazomib (Ninlaro), with lenalidomide and dexamethasone for relapsed or refractory multiple myeloma in adults
Evidence-based recommendations on golimumab (Simponi) for severe non-radiographic axial spondyloarthritis in adults