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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

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

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

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

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

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

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

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

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

Metreleptin for treating lipodystrophy (ID861)

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

Highly specialised technologies guidance In development

Ravulizumab for treating paroxysmal nocturnal haemoglobinuria ID1457

Proposed [GID-HST10023] 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

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

Proposed [GID-HST10020] 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

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

Human alpha1-proteinase inhibitor for treating emphysema ID856

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

Highly specialised technologies guidance In development

Cerliponase alfa for treating neuronal ceroid lipofuscinosis type 2 [ID943]

In development [GID-HST10008] Expected publication date: 27 November 2019

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

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

Atezolizumab with carboplatin and etoposide for untreated extensive-stage small-cell lung cancer ID1504

In development [GID-TA10400] Expected publication date: 11 December 2019

Technology appraisal guidance In development

Transcatheter aortic valve implantation for aortic stenosis (IPG586)

Evidence-based recommendations on transcatheter aortic valve implantation (TAVI) for aortic stenosis

Interventional procedures guidance Published July 2017

Laparoscopic insertion of a magnetic titanium ring for gastro-oesophageal reflux disease (IPG585)

Evidence-based recommendations on laparoscopic insertion of a magnetic titanium ring for gastro-oesophageal reflux disease (GORD) in adults

Interventional procedures guidance Published July 2017

Palliative photodynamic therapy for advanced oesophageal cancer (IPG206)

Evidence-based recommendations on palliative photodynamic therapy (PDT/blue light therapy) for treating advanced oesophageal (gullet) cancer

Interventional procedures guidance Published January 2007

Laparoscopic nephrolithotomy and pyelolithotomy (IPG212)

Evidence-based recommendations on laparoscopic nephrolithotomy and pyelolithotomy for treating renal (kidney) stones

Interventional procedures guidance Published March 2007

Extracorporeal shockwave lithotripsy for calcific tendonitis (tendinopathy) of the shoulder (IPG21)

Evidence-based recommendations on extracorporeal shockwave lithotripsy (ESWT) for calcific tendonitis (tendinopathy) of the shoulder

Interventional procedures guidance Published November 2003

Radiofrequency-assisted liver resection (IPG211)

Evidence-based recommendations on radiofrequency-assisted liver resection for treating primary or secondary liver cancer

Interventional procedures guidance Published February 2007

Laparoscopic distal pancreatectomy (IPG204)

Evidence-based recommendations on laparoscopic distal pancreatectomy for treating pancreatic tumours (benign or malignant) or chronic pancreatitis

Interventional procedures guidance Published January 2007

Fetal cystoscopy for the diagnosis and treatment of lower urinary outflow tract obstruction (IPG205)

Evidence-based recommendations on fetal cystoscopy for diagnosing and treating lower urinary outflow tract blockage in unborn babies

Interventional procedures guidance Published January 2007

Fetal vesico–amniotic shunt for lower urinary tract outflow obstruction (IPG202)

Evidence-based recommendations on fetal vesico–amniotic shunt for treating lower urinary tract outflow blockage in unborn babies

Interventional procedures guidance Published December 2006

Radiofrequency ablation of hepatocellular carcinoma (IPG2)

Evidence-based recommendations on radiofrequency ablation of hepatocellular carcinoma (HCC/liver cancer)

Interventional procedures guidance Published July 2003

Septostomy with or without amnioreduction for the treatment of twin-to-twin transfusion syndrome (IPG199)

Evidence-based recommendations on septostomy with or without amnioreduction for treating twin-to-twin transfusion syndrome (TTTS)

Interventional procedures guidance Published December 2006

Intramedullary distraction for lower limb lengthening (IPG197)

Evidence-based recommendations on intramedullary distraction for lower limb (leg bones) lengthening

Interventional procedures guidance Published December 2006

Patient safety and reduction of risk of transmission of Creutzfeldt–Jakob disease (CJD) via interventional procedures (IPG196)

Evidence-based recommendations on patient safety and reduction of risk of transmission of Creutzfeldt–Jakob disease (CJD) via interventional procedures

Interventional procedures guidance Published November 2006

Therapeutic amnioinfusion for oligohydramnios during pregnancy (excluding labour) (IPG192)

Evidence-based recommendations on therapeutic amnioinfusion for treating oligohydramnios (lack of amniotic fluid ) during pregnancy (excluding labour)

Interventional procedures guidance Published November 2006

Deep brain stimulation for tremor and dystonia (excluding Parkinson's disease) (IPG188)

Evidence-based recommendations on deep brain stimulation (permanent electrodes planted in brain) for tremor and dystonia (excluding Parkinson's disease)

Interventional procedures guidance Published August 2006

Catheterless oesophageal pH monitoring (IPG187)

Evidence-based recommendations on catheterless oesophageal pH monitoring (placing small wireless capsule in gullet to check the level of acid)

Interventional procedures guidance Published July 2006

Tonsillectomy using laser (IPG186)

Evidence-based recommendations on tonsillectomy (removing tonsils) using laser to cut tonsils and seal blood vessels

Interventional procedures guidance Published July 2006

High-intensity focused ultrasound for atrial fibrillation in association with other cardiac surgery (IPG184)

Evidence-based recommendations on high-intensity focused ultrasound for atrial fibrillation (AF) in association with other cardiac surgery

Interventional procedures guidance Published July 2006

Percutaneous laser therapy for fetal tumours (IPG180)

Evidence-based recommendations on percutaneous laser therapy for fetal tumours

Interventional procedures guidance Published June 2006

Tonsillectomy using ultrasonic scalpel (IPG178)

Evidence-based recommendations on tonsillectomy (removing tonsils) using ultrasonic scalpel (high-energy waves to cut tonsils and stop bleeding)

Interventional procedures guidance Published June 2006

Short-term circulatory support with left ventricular assist devices as a bridge to cardiac transplantation or recovery (IPG177)

Evidence-based recommendations on short-term circulatory support with left ventricular assist devices (LVAD) as a bridge to heart transplantation or recovery

Interventional procedures guidance Published June 2006

Percutaneous fetal balloon valvuloplasty for pulmonary atresia with intact ventricular septum (IPG176)

Evidence-based recommendations on percutaneous fetal balloon valvuloplasty for pulmonary atresia with intact ventricular septum (PAIVS)

Interventional procedures guidance Published May 2006

High dose rate brachytherapy in combination with external-beam radiotherapy for localised prostate cancer (IPG174)

Evidence-based recommendations on high dose rate (HDR) brachytherapy in combination with external-beam radiotherapy (EBRT) for localised prostate cancer

Interventional procedures guidance Published May 2006

Laparoscopic helium plasma coagulation for the treatment of endometriosis (IPG171)

Evidence-based recommendations on laparoscopic helium plasma coagulation (plasma beam) for treating endometriosis

Interventional procedures guidance Published May 2006

Soft-palate implants for obstructive sleep apnoea (IPG241)

Evidence-based recommendations on soft-palate implants for treating obstructive sleep apnoea (OSA)

Interventional procedures guidance Published November 2007