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Elosulfase alfa for treating mucopolysaccharidosis type IVa (HST2)

Evidence-based recommendations on elosulfase alfa (Vimizim) for treating mucopolysaccharidosis type IVa in adults and children

Highly specialised technologies guidance Published December 2015

Eculizumab for treating atypical haemolytic uraemic syndrome (HST1)

Evidence-based recommendations on eculizumab (Soliris) for treating atypical haemolytic uraemic syndrome in adults and children

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 in people aged

Highly specialised technologies guidance Published July 2016

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

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

Highly specialised technologies guidance Published February 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

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

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

Highly specialised technologies guidance Published November 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 in people with vision loss caused by inherit

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

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

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: 27 May 2020

Highly specialised technologies guidance In development

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

In development [GID-HST10026] Expected publication date: TBC

Highly specialised technologies guidance In development

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

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

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

OTL-200 for treating metachromatic leukodystrophy [ID1666]

Proposed [GID-HST10028] Expected publication date: TBC

Highly specialised technologies guidance Proposed

Metreleptin for treating lipodystrophy (ID861)

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

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

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

Sacral nerve stimulation for faecal incontinence (IPG99)

Evidence-based recommendations on sacral nerve stimulation for treating faecal incontinence

Interventional procedures guidance Published November 2004

Intraoperative fluorescence angiography for the evaluation of coronary artery bypass graft patency (IPG98)

Evidence-based recommendations on intraoperative fluorescence angiography for evaluating coronary artery bypass graft patency

Interventional procedures guidance Published October 2004

Endovascular closure of patent ductus arteriosus (IPG97)

Evidence-based recommendations on endovascular closure of patent ductus arteriosus (PDA/open blood vessel) by inserting a small blocking device

Interventional procedures guidance Published October 2004

Endovascular closure of atrial septal defect (IPG96)

Evidence-based recommendations on endovascular closure of atrial septal defect (ASD /hole in the heart) by inserting a small blocking device

Interventional procedures guidance Published October 2004

Radiofrequency valvotomy for pulmonary atresia (IPG95)

Evidence-based recommendations on radiofrequency valvotomy for treating pulmonary atresia

Interventional procedures guidance Published October 2004

Intralesional photocoagulation of subcutaneous congenital vascular disorders (IPG90)

Evidence-based recommendations on intralesional photocoagulation for treating subcutaneous congenital vascular disorders

Interventional procedures guidance Published September 2004

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

Endoscopic ablation for a pilonidal sinus (IPG646)

Evidence-based recommendations on endoscopic ablation for a pilonidal sinus in adults

Interventional procedures guidance Published April 2019

Endoscopic ablation for an anal fistula (IPG645)

Evidence-based recommendations on endoscopic ablation for an anal fistula in adults

Interventional procedures guidance Published April 2019

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

High-intensity focused ultrasound for symptomatic benign thyroid nodules (IPG643)

Evidence-based recommendations on high-intensity focused ultrasound for symptomatic benign thyroid nodules in adults

Interventional procedures guidance Published February 2019

Barnett Continent Intestinal Reservoir (modified continent ileostomy) to restore continence after colon and rectum removal (IPG642)

Evidence-based recommendations on Barnett Continent Intestinal Reservoir (modified continent ileostomy) to restore continence after colon and rectum removal

Interventional procedures guidance Published February 2019

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

Sacral nerve stimulation for urge incontinence and urgency-frequency (IPG64)

Evidence-based recommendations on sacral nerve stimulation for treating overactive bladder (urge incontinence and urgency-frequency)

Interventional procedures guidance Published June 2004

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

Ex-situ machine perfusion for extracorporeal preservation of livers for transplantation (IPG636)

Evidence-based recommendations on ex-situ machine perfusion for extracorporeal preservation of livers for transplantation in people of all ages

Interventional procedures guidance Published January 2019

Bronchial thermoplasty for severe asthma (IPG635)

Evidence-based recommendations on bronchial thermoplasty for severe asthma in adults

Interventional procedures guidance Published December 2018

Transcutaneous neuromuscular electrical stimulation for oropharyngeal dysphagia in adults (IPG634)

Evidence-based recommendations on transcutaneous neuromuscular electrical stimulation for oropharyngeal dysphagia (difficulty swallowing) in adults

Interventional procedures guidance Published December 2018

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

Subcutaneous automated low-flow pump implantation for refractory ascites caused by cirrhosis (IPG631)

Evidence-based recommendations on subcutaneous automated low-flow pump implantation for refractory ascites caused by liver cirrhosis in adults

Interventional procedures guidance Published November 2018

Suction diathermy adenoidectomy (IPG328)

Evidence-based recommendations on suction diathermy adenoidectomy for removing adenoids

Interventional procedures guidance Published December 2009