Published: Guidance, quality standards and advice

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Showing 1 to 25 of 53

TitleReference numberPublishedLast updated
Pegzilarginase for treating arginase-1 deficiency in people 2 years and overHST35
Cerliponase alfa for treating neuronal ceroid lipofuscinosis type 2HST34
Leniolisib for treating activated phosphoinositide 3-kinase delta syndrome in people 12 years and overHST33
Burosumab for treating X-linked hypophosphataemia in children and young peopleHST8
Olipudase alfa for treating acid sphingomyelinase deficiency (Niemann–Pick disease) type AB and type BHST32
Urinary tract infection (recurrent): antimicrobial prescribingNG112
Setmelanotide for treating obesity and hyperphagia in Bardet-Biedl syndromeHST31
Sebelipase alfa for treating Wolman diseaseHST30
Velmanase alfa for treating alpha-mannosidosisHST29
Birch bark extract for treating epidermolysis bullosaHST28
Afamelanotide for treating erythropoietic protoporphyriaHST27
Onasemnogene abeparvovec for treating spinal muscular atrophyHST15
Onasemnogene abeparvovec for treating presymptomatic spinal muscular atrophyHST24
Lumasiran for treating primary hyperoxaluria type 1HST25
Eladocagene exuparvovec for treating aromatic L-amino acid decarboxylase deficiencyHST26
Asfotase alfa for treating paediatric-onset hypophosphatasiaHST23
Ataluren for treating Duchenne muscular dystrophy with a nonsense mutation in the dystrophin geneHST22
Ceftazidime with avibactam for treating severe drug-resistant gram-negative bacterial infectionsAMR1
Cefiderocol for treating severe drug-resistant gram-negative bacterial infectionsAMR2
Setmelanotide for treating obesity caused by LEPR or POMC deficiencyHST21
Selumetinib for treating symptomatic and inoperable plexiform neurofibromas associated with type 1 neurofibromatosis in children aged 3 and overHST20
Elosulfase alfa for treating mucopolysaccharidosis type 4AHST19
Atidarsagene autotemcel for treating metachromatic leukodystrophyHST18
Otitis media (acute): antimicrobial prescribingNG91
Odevixibat for treating progressive familial intrahepatic cholestasisHST17

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