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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.
NICE is unable to make a recommendation on ruxolitinib (Jakavi) for treating moderate to severe chronic graft-versus-host disease after an allogeneic stem cell transplant in people 28 days to 17 years. This is because the company did not provide an evidence submission.
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Sections for TA1132
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.
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.
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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Sections for TA1135
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.
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.
Evidence-based recommendations on tisagenlecleucel (Kymriah) for treating relapsed or refractory B-cell acute lymphoblastic leukaemia in people 25 years and under.
Evidence-based recommendations on pembrolizumab (Keytruda) with paclitaxel or nab-paclitaxel for triple-negative, locally recurrent unresectable or metastatic breast cancer in adults who have not had chemotherapy for metastatic disease.
Evidence-based recommendations on nivolumab (Opdivo) as a possible treatment for completely resected oesophageal or gastro-oesophageal junction cancer in adults.
Evidence-based recommendations on anakinra (Kineret) for treating adult-onset Still’s disease and systemic juvenile idiopathic arthritis in people 8 months and older.
NICE is unable to make a recommendation on tabelecleucel (Ebvallo) for treating post-transplant lymphoproliferative disorder caused by the Epstein-Barr virus. This is because Pierre Fabre Ltd did not provide an evidence submission. We will review this decision if the company decides to make a submission.
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Sections for TA923
Evidence-based recommendations on glofitamab (Columvi) for treating relapsed or refractory diffuse large B-cell lymphoma in adults after 2 or more systemic treatments.
Evidence-based recommendations on using dual-chamber pacemakers for symptomatic bradycardia due to sick sinus syndrome or atrioventricular block.