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Showing 1 to 10 of 12
Title Type Expected publication date
Afamelanotide for treating erythropoietic protoporphyria [ID927] Highly specialised technology Expected publication date:TBC 0
Burosumab for treating X-linked hypophosphataemia [ID1151] Highly specialised technology Expected publication date:24 October 2018 20181024
Cerliponase alfa for treating neuronal ceroid lipofuscinosis type 2 [ID943] Highly specialised technology Expected publication date:TBC 0
Drisapersen for the first-line treatment of Duchenne’s muscular dystrophy [ID911] Highly specialised technology Expected publication date:TBC 0
Eteplirsen for treating Duchenne muscular dystrophy [ID1003] Highly specialised technology Expected publication date:TBC 0
Human alpha1-proteinase inhibitor for treating emphysema ID856 Highly specialised technology Expected publication date:13 February 2019 20190213
Inotersen for treating hereditary transthyretin-related amyloidosis [ID1242] Highly specialised technology Expected publication date:TBC 0
Lysosomal acid lipase deficiency - sebelipase alfa [ID737] Highly specialised technology Expected publication date:TBC 0
Metreleptin for treating lipodystrophy (ID861) Highly specialised technology Expected publication date:26 September 2018 20180926
Patisiran for treating hereditary transthyretin-related amyloidosis [ID1279] Highly specialised technology Expected publication date:TBC 0