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Elosulfase alfa for treating mucopolysaccharidosis type 4A (HST19)
Evidence-based recommendations on elosulfase alfa (Vimizim) for treating mucopolysaccharidosis type 4A in people of all ages.
Evidence-based recommendations on selumetinib (Koselugo) for treating symptomatic and inoperable plexiform neurofibromas associated with type 1 neurofibromatosis in children aged 3 and over.
Afamelanotide for treating erythropoietic protoporphyria (HST27)
Evidence-based recommendations on afamelanotide (Scenesse) for treating erythropoietic protoporphyria in adults.
Evidence-based recommendations on velmanase alfa (Lamzede) for treating alpha-mannosidosis in people under 18 years and in people who turn 18 while on treatment.
Evidence-based recommendations on migalastat (Galafold) for treating Fabry disease in people over 16.
Evidence-based recommendations on eliglustat (Cerdelga) for treating type 1 Gaucher disease in adults.
Strimvelis for treating adenosine deaminase deficiency–severe combined immunodeficiency (HST7)
Evidence-based recommendation on Strimvelis for adenosine deaminase deficiency–severe combined immunodeficiency.
Inotersen for treating hereditary transthyretin amyloidosis (HST9)
Evidence-based recommendations on inotersen (Tegsedi) for stage 1 and stage 2 polyneuropathy in adults with hereditary transthyretin amyloidosis.
Evidence-based recommendations on tafasitamab (Minjuvi) with lenolidomide for relapsed or refractory diffuse large B-cell lymphoma in adults who cannot have an autologous stem cell transplant.
Evidence-based recommendations on olaparib (Lynparza) for adjuvant treatment of BRCA mutation-positive HER2-negative high-risk early breast cancer after chemotherapy in adults.
Difelikefalin for treating pruritus in people having haemodialysis (TA890)
Evidence-based recommendations on difelikefalin (Kapruvia) for pruritus in adults with chronic kidney disease having haemodialysis.
Ibrutinib with venetoclax for untreated chronic lymphocytic leukaemia (TA891)
Evidence-based recommendations on ibrutinib (Imbruvica) with venetoclax (Venclyxto) for untreated chronic lymphocytic leukaemia in adults.
Venetoclax with obinutuzumab for untreated chronic lymphocytic leukaemia (TA663)
Evidence-based recommendations on venetoclax (Venclyxto) with obinutuzumab for untreated chronic lymphocytic leukaemia in adults.
Evidence-based recommendations on risdiplam (Evrysdi) for 5q spinal muscular atrophy (SMA) in people of all ages.
Deucravacitinib for treating moderate to severe plaque psoriasis (TA907)
Evidence-based recommendations on deucravacitinib for treating moderate to severe plaque psoriasis in adults.