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Asfotase alfa for treating paediatric-onset hypophosphatasia (HST6)

Evidence-based recommendations on asfotase alfa (Strensiq) for treating paediatric-onset hypophosphatasia in adults and children

Highly specialised technologies guidance Published August 2017

Eliglustat for treating type 1 Gaucher disease (HST5)

Evidence-based recommendations on eliglustat (Cerdelga) for treating type 1 Gaucher disease in adults

Highly specialised technologies guidance Published June 2017

Migalastat for treating Fabry disease (HST4)

Evidence-based recommendations on migalastat (Galafold) for treating Fabry disease in people over 16

Highly specialised technologies guidance Published February 2017

Ataluren for treating Duchenne muscular dystrophy with a nonsense mutation in the dystrophin gene (HST3)

Evidence-based recommendations on ataluren (Translarna) for treating Duchenne muscular dystrophy with a nonsense mutation in the dystrophin gene..

Highly specialised technologies guidance Published July 2016

Elosulfase alfa for treating mucopolysaccharidosis type IVa (HST2)

Evidence-based recommendations on elosulfase alfa (Vimizim) for treating mucopolysaccharidosis type IVa (MPS IVA)

Highly specialised technologies guidance Published December 2015

Voretigene neparvovec for treating inherited retinal dystrophies caused by RPE65 gene mutations (HST11)

Evidence-based recommendations on voretigene neparvovec (Luxturna) for RPE65-mediated inherited retinal dystrophies (confirmed biallelic RPE65 gene mutations)

Highly specialised technologies guidance Published October 2019

Patisiran for treating hereditary transthyretin amyloidosis (HST10)

Evidence-based recommendations on patisiran (Onpattro) for treating hereditary transthyretin amyloidosis in adults with stage 1 and stage 2 polyneuropathy

Highly specialised technologies guidance Published August 2019

Eculizumab for treating atypical haemolytic uraemic syndrome (HST1)

Evidence-based recommendations on culizumab (Soliris) for treating atypical haemolytic uraemic syndrome (aHUS)

Highly specialised technologies guidance Published January 2015

Inotersen for treating hereditary transthyretin amyloidosis (HST9)

Evidence-based recommendations on inotersen (Tegsedi) for stage 1 and stage 2 polyneuropathy in adults with hereditary transthyretin amyloidosis

Highly specialised technologies guidance Published May 2019

Burosumab for treating X-linked hypophosphataemia in children and young people (HST8)

Evidence-based recommendations on burosumab (Crysvita) for treating X-linked hypophosphataemia (XLH) in children and young people

Highly specialised technologies guidance Published October 2018

Strimvelis for treating adenosine deaminase deficiency–severe combined immunodeficiency (HST7)

Evidence-based recommendation on Strimvelis for adenosine deaminase deficiency–severe combined immunodeficiency (ADA–SCID)

Highly specialised technologies guidance Published February 2018

Cerliponase alfa for treating neuronal ceroid lipofuscinosis type 2 (HST12)

Evidence-based recommendations on cerliponase alfa (Brineura) for treating neuronal ceroid lipofuscinosis type 2 (CLN2) in children

Highly specialised technologies guidance Published November 2019

Afamelanotide for treating erythropoietic protoporphyria [ID927]

In development [GID-HST10009] Expected publication date: TBC

Highly specialised technologies guidance In development

VTS-270 for treating Niemann-Pick type C1 (ID1267)

Proposed [GID-HST10020] Expected publication date: TBC

Highly specialised technologies guidance Proposed

OTL-101 for treating adenosine deaminase deficiency–severe combined immunodeficiency [ID1152]

Proposed [GID-HST10022] Expected publication date: TBC

Highly specialised technologies guidance Proposed

Ravulizumab for treating paroxysmal nocturnal haemoglobinuria ID1457

Proposed [GID-HST10023] Expected publication date: TBC

Highly specialised technologies guidance Proposed

Velmanase alfa for treating alpha-mannosidosis [ID800]

In development [GID-HST10010] Expected publication date: TBC

Highly specialised technologies guidance In development

Onasemnogene abeparvovec for treating spinal muscular atrophy type 1 [ID1473]

In development [GID-HST10026] Expected publication date: 08 September 2020

Highly specialised technologies guidance In development

Lysosomal acid lipase deficiency - sebelipase alfa [ID737]

In development [GID-LYSOSOMALACIDLIPASEDEFICIENCYSEBELIPASEALFAID737] Expected publication date: TBC

Highly specialised technologies guidance In development

Intrathecal idursulfase for treating Mucopolysaccharidosis type II ID1223

Proposed [GID-HST10019] Expected publication date: TBC

Highly specialised technologies guidance Proposed

OTL-200 for treating metachromatic leukodystrophy [ID1666]

Proposed [GID-HST10028] Expected publication date: TBC

Highly specialised technologies guidance Proposed

Elosulfase alfa for treating mucopolysaccharidosis type IVa (review of HST2) [ID1643]

In development [GID-HST10027] Expected publication date: 11 November 2020

Highly specialised technologies guidance In development

Volanesorsen for treating familial chylomicronaemia syndrome [ID1326]

In development [GID-HST10015] Expected publication date: 03 June 2020

Highly specialised technologies guidance In development

Human alpha1-proteinase inhibitor for treating emphysema ID856

In development [GID-HST10017] Expected publication date: TBC

Highly specialised technologies guidance In development

Metreleptin for treating lipodystrophy (ID861)

In development [GID-HST10011] Expected publication date: 13 January 2021

Highly specialised technologies guidance In development

Volanesorsen for treating familial chylomicronaemia syndrome [ID1326]: evaluation consultation

We are listening to your views on this highly specialised technology. Comments close 27 January 2020.

Highly specialised technologies guidance In consultation

Eteplirsen for treating Duchenne muscular dystrophy [ID1003]

In development [GID-HST10007] Expected publication date: TBC

Highly specialised technologies guidance In development

Drisapersen for the first-line treatment of Duchenne's muscular dystrophy [ID911]

In development [GID-HST10004] Expected publication date: TBC

Highly specialised technologies guidance In development

Joint distraction for ankle osteoarthritis (IPG538)

Evidence-based recommendations on joint distraction for ankle osteoarthritis

Interventional procedures guidance Published December 2015

Insertion of a subretinal prosthesis system for retinitis pigmentosa (IPG537)

Evidence-based recommendations on insertion of a subretinal prosthesis system for retinitis pigmentosa (RP)

Interventional procedures guidance Published December 2015

Sacral nerve stimulation for idiopathic chronic non-obstructive urinary retention (IPG536)

Evidence-based recommendations on sacral nerve stimulation for idiopathic chronic non-obstructive urinary retention

Interventional procedures guidance Published November 2015

Living-donor liver transplantation (IPG535)

Evidence-based recommendations on living-donor liver transplantation

Interventional procedures guidance Published November 2015

Implantation of a corneal graft–keratoprosthesis for severe corneal opacity in wet blinking eyes (IPG534)

Evidence-based recommendations on implantation of a corneal graft–keratoprosthesis for severe corneal opacity in wet blinking eyes

Interventional procedures guidance Published November 2015

Implanting a baroreceptor stimulation device for resistant hypertension (IPG533)

Evidence-based recommendations on implanting a baroreceptor stimulation device for resistant hypertension

Interventional procedures guidance Published October 2015

Low energy contact X-ray brachytherapy (the Papillon technique) for early stage rectal cancer (IPG532)

Evidence-based recommendations on low energy contact X-ray brachytherapy (the Papillon technique) for treating early stage rectal cancer

Interventional procedures guidance Published September 2015

Preoperative high dose rate brachytherapy for rectal cancer (IPG531)

Evidence-based recommendations on preoperative high dose rate brachytherapy for treating rectal cancer

Interventional procedures guidance Published August 2015

Transcranial direct current stimulation (tDCS) for depression (IPG530)

Evidence-based recommendations on transcranial direct current stimulation (tDCS) for depression

Interventional procedures guidance Published August 2015

Computed tomography-guided thermocoagulation of osteoid osteoma (IPG53)

Evidence-based recommendations on CT-guided thermocoagulation for osteoid osteoma (non-cancerous tumours/growths of bone tissue)

Interventional procedures guidance Published March 2004

Joint distraction for knee osteoarthritis without alignment correction (IPG529)

Evidence-based recommendations on joint distraction for knee osteoarthritis without alignment correction

Interventional procedures guidance Published July 2015

Insertion of a double balloon catheter for induction of labour in pregnant women without previous caesarean section (IPG528)

Evidence-based recommendations on the insertion of a double balloon catheter for inducing labour in pregnant women without previous caesarean section

Interventional procedures guidance Published July 2015

Implantation of a sphenopalatine ganglion stimulation device for chronic cluster headache (IPG527)

Evidence-based recommendations on the implantation of a sphenopalatine ganglion stimulation device for treating chronic cluster headaches

Interventional procedures guidance Published June 2015

Cyanoacrylate glue occlusion for varicose veins (IPG526)

Evidence-based recommendations on cyanoacrylate glue occlusion for varicose veins

Interventional procedures guidance Published June 2015

Electrotherapy for the treatment of haemorrhoids (IPG525)

Evidence-based recommendations on electrotherapy for treating haemorrhoids

Interventional procedures guidance Published June 2015

Hysteroscopic morcellation of uterine leiomyomas (fibroids) (IPG522)

Evidence-based recommendations on hysteroscopic morcellation of uterine leiomyomas (uterine myomas or fibroids)

Interventional procedures guidance Published June 2015

Suture fixation of acute disruption of the distal tibiofibular syndesmosis (IPG521)

Evidence-based recommendations on suture fixation of acute disruption of the distal tibiofibular syndesmosis (ligament injuries)

Interventional procedures guidance Published June 2015

Radiation therapy for early Dupuytren's disease (IPG573)

Evidence-based recommendations on radiation therapy (low energy X-rays) for treating early Dupuytren’s contractures in adults

Interventional procedures guidance Published December 2016

Irreversible electroporation for treating prostate cancer (IPG572)

Evidence-based recommendations on electroporation (electrical pulses sent through a needle into a tumour) for treating prostate cancer in adults

Interventional procedures guidance Published December 2016

Extracorporeal shockwave therapy for Achilles tendinopathy (IPG571)

Evidence-based recommendations on extracorporeal shockwave therapy for treating Achilles tendinopathy in adults

Interventional procedures guidance Published December 2016

Epiduroscopic lumbar discectomy through the sacral hiatus for sciatica (IPG570)

Evidence-based recommendations on epiduroscopic lumbar discectomy through the sacral hiatus for sciatica in adults

Interventional procedures guidance Published December 2016

Laparoscopic live donor simple nephrectomy (IPG57)

Evidence-based recommendations on laparoscopic live donor simple nephrectomy (keyhole surgery to remove kidney from donor)

Interventional procedures guidance Published May 2004