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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

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

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

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

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

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

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

Intrathecal idursulfase for treating Mucopolysaccharidosis type II ID1223

Proposed [GID-HST10019] Expected publication date: TBC

Highly specialised technologies guidance Proposed

Metreleptin for treating lipodystrophy (ID861)

In development [GID-HST10011] Expected publication date: TBC

Highly specialised technologies guidance In development

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

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

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

Volanesorsen for treating familial chylomicronaemia syndrome [ID1326]

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

Human alpha1-proteinase inhibitor for treating emphysema ID856

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

Highly specialised technologies guidance In development

Velmanase alfa for treating alpha-mannosidosis [ID800]

In development [GID-HST10010] 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

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

Eteplirsen for treating Duchenne muscular dystrophy [ID1003]

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

Highly specialised technologies guidance In development

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

Reinforcement of a permanent stoma with a synthetic or biological mesh to prevent a parastomal hernia (IPG654)

Evidence-based recommendations on reinforcement of a permanent stoma with a synthetic or biological mesh to prevent a parastomal hernia

Interventional procedures guidance Published June 2019

Percutaneous mitral valve leaflet repair for mitral regurgitation (IPG649)

Evidence-based recommendations on percutaneous mitral valve leaflet repair for mitral regurgitation in adults

Interventional procedures guidance Published May 2019

Therapeutic hypothermia for acute ischaemic stroke (IPG647)

Evidence-based recommendations on therapeutic hypothermia (using a cooling device to reduce the body’s temperature) for acute ischaemic stroke in adults

Interventional procedures guidance Published May 2019

Implant insertion for prominent ears (IPG660)

Evidence-based recommendations on implant insertion for prominent ears in children, young people and adults

Interventional procedures guidance Published September 2019

Bioprosthetic plug insertion for anal fistula (IPG662)

Evidence-based recommendations on bioprosthetic plug insertion for anal fistula in adults

Interventional procedures guidance Published September 2019

High-intensity focused ultrasound for glaucoma (IPG661)

Evidence-based recommendations on high-intensity focused ultrasound for glaucoma in adults

Interventional procedures guidance Published September 2019

Endovascular insertion of an intrasaccular wire-mesh blood-flow disruption device for intracranial aneurysms (IPG658)

Evidence-based recommendations on endovascular insertion of an intrasaccular wire-mesh blood-flow disruption device for intracranial aneurysms in adults

Interventional procedures guidance Published August 2019

Complete cytoreduction for pseudomyxoma peritonei (Sugarbaker technique) (IPG56)

Evidence-based recommendations on complete cytoreduction (Sugarbaker technique) for pseudomyxoma peritonei (abnormal tumour that starts in appendix/bowel)

Interventional procedures guidance Published April 2004

Extracorporeal carbon dioxide removal for acute respiratory failure (IPG564)

Evidence-based recommendations on extracorporeal carbon dioxide removal for acute respiratory failure in adults

Interventional procedures guidance Published August 2016

Transcervical extracorporeal reverse flow neuroprotection for reducing the risk of stroke during carotid artery stenting (IPG561)

Evidence-based recommendations on transcervical extracorporeal reverse flow neuroprotection for reducing the risk of stroke during carotid artery stenting

Interventional procedures guidance Published June 2016

Endoscopic transluminal pancreatic necrosectomy (IPG567)

Evidence-based recommendations on endoscopic transluminal pancreatic necrosectomy for treating pancreatic necrosis (necrotising pancreatitis) in adults

Interventional procedures guidance Published November 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

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