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Evidence-based recommendations on sebelipase alfa (Kanuma) for long-term enzyme replacement therapy in Wolman disease (rapidly progressive lysosomal acid lipase deficiency) in people aged 2 years and under when treatment starts.
Cerliponase alfa for treating neuronal ceroid lipofuscinosis type 2 (HST34)
Evidence-based recommendations on cerliponase alfa (Brineura) for treating neuronal ceroid lipofuscinosis type 2 in children.
Evidence-based recommendations on belantamab mafodotin (Blenrep) with pomalidomide and dexamethasone for previously treated multiple myeloma in adults.
Dupilumab for treating severe chronic rhinosinusitis with nasal polyps (TA1134)
Evidence-based recommendations on dupilumab (Dupixent) for treating severe chronic rhinosinusitis with nasal polyps in adults.
NICE is unable to make a recommendation on baloxavir marboxil (Xofluza) for treating and preventing influenza in children 1 to 11 years. This is because the company did not provide an evidence submission.
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Holoclar for treating limbal stem cell deficiency after eye burns (TA467)
Evidence-based recommendations on Holoclar (a layer of cells grown from cells taken from your own eye) for treating limbal stem cell deficiency in adults with eye burns.
Setmelanotide for treating obesity and hyperphagia in Bardet-Biedl syndrome (HST31)
Evidence-based recommendations on setmelanotide (Imcivree) for treating obesity and hyperphagia in Bardet-Biedl syndrome in people 6 years and over, only if they are between 6 and 17 years when treatment starts.
Burosumab for treating X-linked hypophosphataemia in children and young people (HST8)
Evidence-based recommendations on burosumab (Crysvita) for X-linked hypophosphataemia in children and young people.
Belzutifan for treating tumours associated with von Hippel-Lindau disease (TA1011)
Evidence-based recommendations on belzutifan (Welireg) for treating tumours associated with von Hippel-Lindau disease in adults.
Eladocagene exuparvovec for treating aromatic L-amino acid decarboxylase deficiency (HST26)
Evidence-based recommendations on eladocagene exuparvovec (Upstaza) for treating aromatic L-amino acid decarboxylase deficiency in people 18 months and over.
Evidence-based recommendations on osimertinib (Tagrisso) for adjuvant treatment of stage 1b to 3a non-small-cell lung cancer after complete tumour resection in adults.
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Evidence-based recommendations on ceritinib (Zykadia) for treating advanced anaplastic lymphoma kinase positive non-small-cell lung cancer in adults who have had crizotinib.
Evidence-based recommendations on vemurafenib (Zelboraf) for treating advanced BRAF V600-positive malignant melanoma in adults.
Evidence-based recommendations on ruxolitinib (Jakavi). This drug is for adults with disease-related splenomegaly or symptoms caused by primary myelofibrosis (also known as chronic idiopathic myelofibrosis), post polycythaemia vera myelofibrosis or post essential thrombocythaemia myelofibrosis, only if they have intermediate-2 or high-risk disease.
Aflibercept solution for injection for treating wet age‑related macular degeneration (TA294)
Evidence-based recommendations on aflibercept solution for injection (Eylea) for treating wet age-related macular degeneration in adults.