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Onasemnogene abeparvovec for treating presymptomatic spinal muscular atrophy (HST24)
Evidence-based recommendations on onasemnogene abeparvovec (Zolgensma) for treating presymptomatic spinal muscular atrophy in babies aged 12 months and under.
Evidence-based recommendations on lumasiran (Oxlumo) for primary hyperoxaluria type 1 in people of all ages.
Evidence-based recommendations on givosiran (Givlaari) for treating acute hepatic porphyria in adults and young people aged 12 and over.
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.
Atidarsagene autotemcel for treating metachromatic leukodystrophy (HST18)
Evidence-based recommendations on atidarsagene autotemcel (Libmeldy) for treating metachromatic leukodystrophy in children.
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.
Selpercatinib for treating advanced thyroid cancer with RET alterations (TA742)
Evidence-based recommendations on selpercatinib (Retsevmo) for advanced thyroid cancer with RET alterations in people 12 years and older.