Date published | Highly Specialised Technology Guidance |
January 2015 | HST1 - Eculizumab (Soliris) for treating atypical haemolytic uraemic syndrome |
December 2015 | HST2 - Elosulfase alfa (Vimizim) for treating mucopolysaccharidosis type IVa |
July 2016 | HST3 - Ataluren for treating Duchenne muscular dystrophy with a nonsense mutation in the dystrophin gene |
February 2017 | HST4 - Migalastat (Galafold) for treating Fabry disease |
June 2017 | HST5 - Eliglustat (Cerdelga) for treating type 1 Gaucher disease |
August 2017 | HST6 - Asfotase (Strensiq) alfa for treating paediatric-onset hypophosphatasia |
February 2018 | HST7 - Strimvelis for treating adenosine deaminase deficiency-severe combined immunodeficiency |
October 2018 | HST8 - Burosumab (Crysvita) for treating X-linked hypophosphataemia in children and young people |
May 2019 | HST9 - Inotersen (Tegsedi) for treating hereditary transthyretin amyloidosis |
August 2019 | HST10 - Patisiran (Onpattro) for treating hereditary transthyretin amyloidosis |
November 2019 | HST11 - Voretigene neparvovec (Luxturna) - for treating inherited retinal dystrophies caused by RPE65 gene mutations |
December 2019 | HST12 - Cerliponase alfa for treating neuronal ceroid lipofuscinosis type 2 |
November 2020 | HST13 - Volanesorsen for treating familial chylomicronaemia syndrome |
March 2021 | HST14 - Metreleptin for treating lipodystrophy |
July 2021 | HST15 - Onasemnogene abeparvovec for treating spinal muscular atrophy *[updated April 2023] |
November 2021 | HST16 - Givosiran for treating acute hepatic porphyria |
February 2022 | HST17 - Odevixibat for treating progressive familial intrahepatic cholestasis |
March 2022 | HST18 - Atidarsagene autoemcel for treating metachromatic leukodystrophy |
April 2022 | HST19 - Elosulfase alfa for treating mucopolysaccharidosis type 4A |
May 2022 | HST20 - Selumetinib for treating symptomatic and inoperable plexiform neurofibroma associated with type 1 neurofibromatosis in children aged 3 and over |
July 2022 | HST21 - Setmelanotide for treating obesituy caused by LEPR or POMC deficiency |
February 2023 | HST22 - Ataluren for treating Duchenne muscular dystrophy with a nonsense mutation in the dystrophin gene |
March 2023 | HST23 - Asfotase alfa for treating paediatric-onset hypophosphatasia |
April 2023 *updated | HST15 -Onasemnogene abeparvovec for treating spinal muscular atrophy |
April 2023 | HST24 - Onasemnogene abeparvovec for treating presymptomatic spinal muscular atrophy |
April 2023 | HST25 - Lumasiran for treating primary hyperoxaluria type 1 |
April 2023 | HST26 - Eladocagene exuparvovec for treating aromatic L-amino acid decarboxylase deficiency |
July 2023 | HST27 - Afamelanotide for treating erythropoietic protoporphyria |
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