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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

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

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

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

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

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

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

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

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

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

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

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: 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

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

Intrathecal idursulfase for treating Mucopolysaccharidosis type II ID1223

Proposed [GID-HST10019] Expected publication date: TBC

Highly specialised technologies guidance Proposed

Afamelanotide for treating erythropoietic protoporphyria [ID927]

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

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

OTL-200 for treating metachromatic leukodystrophy [ID1666]

Proposed [GID-HST10028] 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

Volanesorsen for treating familial chylomicronaemia syndrome [ID1326]

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

Highly specialised technologies guidance In development

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

In development [GID-HST10027] Expected publication date: TBC

Highly specialised technologies guidance In development

Cyanoacrylate glue occlusion for varicose veins (IPG526)

Evidence-based recommendations on cyanoacrylate glue occlusion for varicose veins

Interventional procedures guidance Published June 2015

Prostatic urethral temporary implant insertion for lower urinary tract symptoms caused by benign prostatic hyperplasia (IPG641)

Evidence-based recommendations on prostatic urethral temporary implant insertion for lower urinary tract symptoms caused by benign prostatic hyperplasia

Interventional procedures guidance Published January 2019

Percutaneous venoplasty for chronic cerebrospinal venous insufficiency in multiple sclerosis (IPG640)

Evidence-based recommendations on percutaneous venoplasty for chronic cerebrospinal venous insufficiency (CCSVI) in people with multiple sclerosis (MS)

Interventional procedures guidance Published January 2019

Laparoscopic cerclage for cervical incompetence to prevent late miscarriage or preterm birth (IPG639)

Evidence-based recommendations on laparoscopic cerclage for cervical incompetence to prevent late miscarriage or preterm birth

Interventional procedures guidance Published January 2019

Percutaneous insertion of a temporary heart pump for left ventricular haemodynamic support in high-risk percutaneous coronary interventions (IPG633)

Evidence-based recommendations on percutaneous insertion of a temporary heart pump for left ventricular haemodynamic support in high-risk PCIs

Interventional procedures guidance Published November 2018

Transcranial MRI-guided focused ultrasound thalamotomy for neuropathic pain (IPG632)

Evidence-based recommendations on transcranial MRI-guided focused ultrasound thalamotomy for neuropathic pain in adults

Interventional procedures guidance Published November 2018

Bronchoscopic thermal vapour ablation for upper-lobe emphysema (IPG652)

Evidence-based recommendations on bronchoscopic thermal vapour ablation for upper-lobe emphysema in adults

Interventional procedures guidance Published June 2019

Percutaneous insertion of a cerebral protection device to prevent cerebral embolism during TAVI (IPG650)

Evidence-based recommendations on percutaneous insertion of a cerebral protection device to prevent cerebral embolism during TAVI (transcatheter aortic valve

Interventional procedures guidance Published June 2019

Valve-in-valve TAVI for aortic bioprosthetic valve dysfunction (IPG653)

Evidence-based recommendations on valve-in-valve transcatheter aortic valve implantation (ViV-TAVI) for aortic bioprosthetic valve dysfunction in adults

Interventional procedures guidance Published June 2019

Ultrasound-guided high-intensity transcutaneous focused ultrasound for symptomatic uterine fibroids (IPG657)

Evidence-based recommendations on ultrasound-guided high-intensity transcutaneous focused ultrasound for symptomatic uterine fibroids in adults

Interventional procedures guidance Published July 2019

Transurethral laser ablation for recurrent non-muscle-invasive bladder cancer (IPG656)

Evidence-based recommendations on transurethral laser ablation for recurrent non-muscle-invasive bladder cancer in adults

Interventional procedures guidance Published July 2019

Hypoglossal nerve stimulation for moderate to severe obstructive sleep apnoea (IPG598)

Evidence-based recommendations on hypoglossal nerve stimulation for moderate to severe obstructive sleep apnoea in adults

Interventional procedures guidance Published November 2017

Processed nerve allografts to repair peripheral nerve discontinuities (IPG597)

Evidence-based recommendations on processed nerve allografts (specially treated nerves) to repair peripheral nerve discontinuities (bridge gaps)

Interventional procedures guidance Published November 2017

Radially emitting laser fibre treatment of an anal fistula (IPG644)

Evidence-based recommendations on radially emitting laser fibre treatment of an anal fistula in adults

Interventional procedures guidance Published March 2019

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

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

Living-donor liver transplantation (IPG535)

Evidence-based recommendations on living-donor liver transplantation

Interventional procedures guidance Published November 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

Joint distraction for ankle osteoarthritis (IPG538)

Evidence-based recommendations on joint distraction for ankle osteoarthritis

Interventional procedures guidance Published December 2015

Percutaneous coblation of the intervertebral disc for low back pain and sciatica (IPG543)

Evidence-based recommendations on percutaneous coblation of the intervertebral disc for low back pain and sciatica

Interventional procedures guidance Published January 2016

Normothermic extracorporeal preservation of hearts for transplantation following donation after brainstem death (IPG549)

Evidence-based recommendations on normothermic extracorporeal heart preservation for transplantation following donation after brainstem death

Interventional procedures guidance Published February 2016

Corticosteroid-eluting bioabsorbable stent or spacer insertion during endoscopic sinus surgery to treat chronic rhinosinusitis (IPG551)

Evidence-based recommendations on corticosteroid-eluting bioabsorbable stent or spacer insertion during endoscopic sinus surgery for chronic rhinosinusitis

Interventional procedures guidance Published March 2016

Percutaneous interlaminar endoscopic lumbar discectomy for sciatica (IPG555)

Evidence-based recommendations on percutaneous interlaminar endoscopic lumbar discectomy for treating sciatica

Interventional procedures guidance Published April 2016

Laparoscopic gastric plication for the treatment of severe obesity (IPG432)

Evidence-based recommendations on laparoscopic gastric plication for treating severe obesity

Interventional procedures guidance Published November 2012

Endoscopic radiofrequency ablation for gastro-oesophageal reflux disease (IPG461)

Evidence-based recommendations on endoscopic radiofrequency ablation for gastro-oesophageal reflux disease (acid reflux/ GORD/GERD)

Interventional procedures guidance Published August 2013