Zanubrutinib for treating Waldenström's macroglobulinaemia [ID1427]: draft guidance
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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 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 givosiran (Givlaari) for treating acute hepatic porphyria in adults and young people aged 12 and over
Evidence-based recommendations on elosulfase alfa (Vimizim) for treating mucopolysaccharidosis type 4A in people of all ages
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 odevixibat (Bylvay) for treating progressive familial intrahepatic cholestasis in people 6 months and older
Evidence-based recommendations on atidarsagene autotemcel (Libmeldy) for treating metachromatic leukodystrophy in children
Evidence-based recommendations on migalastat (Galafold) for treating Fabry disease in people over 16
Evidence-based recommendations on ranibizumab (Lucentis) for treating sight problems caused by macular oedema secondary to retinal vein occlusion
NICE is unable to recommend the use in the NHS of canakinumab for treating gouty arthritis attacks and reducing the frequency of subsequent attacks
Evidence-based recommendations on percutaneous vertebroplasty and percutaneous balloon kyphoplasty for osteoporotic vertebral compression fractures
Evidence-based recommendations on colistimethate sodium and tobramycin dry powders for inhalation for pseudomonas lung infection in cystic fibrosis
Evidence-based recommendations on apixaban (Eliquis) for preventing stroke and systemic embolism in adults with non-valvular atrial fibrillation
Evidence-based recommendations on ranibizumab (Lucentis) for treating diabetic macular oedema
Evidence-based recommendations on talimogene laherparepvec (Imlygic) for treating unresectable metastatic melanoma in adults when systemically administered
NICE is unable to make a recommendation on recombinant human parathyroid hormone for treating hypoparathyroidism because Shire Pharmaceuticals
Evidence based recommendations on patiromer (Veltassa) for treating hyperkalaemia in adults
Evidence-based recommendations on sotagliflozin with insulin for treating type 1 diabetes in adults with a body mass index (BMI) of at least 27 kg/m2
Evidence-based recommendations on palbociclib (Ibrance) with fulvestrant for hormone receptor-positive, HER2-negative locally advanced/metastatic breast cancer
Evidence-based recommendations on Palforzia for treating peanut allergy in children and young people
Evidence-based recommendations on fremanezumab (Ajovy) for preventing migraine in adults
Evidence-based recommendations on glecaprevir–pibrentasvir (Maviret) for treating chronic hepatitis C in adults
Evidence-based recommendations on ustekinumab (Stelara) for previously treated moderately to severely active Crohn’s disease 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