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Showing 1 to 10 of 11
Title Type Expected publication date
Afamelanotide for treating erythropoietic protoporphyria [ID927] Highly specialised technology Expected publication date:TBC 0
Cerliponase alfa for treating neuronal ceroid lipofuscinosis type 2 [ID943] Highly specialised technology Expected publication date:27 November 2019 20191127
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: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:TBC 0
Onasemnogene abeparvovec for treating spinal muscular atrophy type 1 [ID1473] Highly specialised technology Expected publication date:03 June 2020 20200603
Velmanase alfa for treating alpha-mannosidosis [ID800] Highly specialised technology Expected publication date:TBC 0
Volanesorsen for treating familial chylomicronaemia syndrome [ID1326] Highly specialised technology Expected publication date:TBC 0