Published: Guidance, quality standards and advice

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Showing 1 to 10 of 30

TitleReference numberPublishedLast updated
Afamelanotide for treating erythropoietic protoporphyriaHST27
Asfotase alfa for treating paediatric-onset hypophosphatasiaHST23
Ataluren for treating Duchenne muscular dystrophy with a nonsense mutation in the dystrophin geneHST22
Atidarsagene autotemcel for treating metachromatic leukodystrophyHST18
Birch bark extract for treating epidermolysis bullosaHST28
Burosumab for treating X-linked hypophosphataemia in children and young peopleHST8
Cerliponase alfa for treating neuronal ceroid lipofuscinosis type 2HST12
Eculizumab for treating atypical haemolytic uraemic syndromeHST1
Eladocagene exuparvovec for treating aromatic L-amino acid decarboxylase deficiencyHST26
Eliglustat for treating type 1 Gaucher diseaseHST5

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