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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 in people with vision loss caused by inherit

Highly specialised technologies guidance Published October 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

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

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

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

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

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

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

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 eculizumab (Soliris) for treating atypical haemolytic uraemic syndrome in adults and children

Highly specialised technologies guidance Published January 2015

OTL-200 for treating metachromatic leukodystrophy [ID1666]

Proposed [GID-HST10028] Expected publication date: TBC

Highly specialised technologies guidance Proposed

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

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

Proposed [GID-HST10022] 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

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

Proposed [GID-HST10020] 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

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

In development [GID-HST10026] 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

Ravulizumab for treating paroxysmal nocturnal haemoglobinuria ID1457

Proposed [GID-HST10023] 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

Leukapheresis for inflammatory bowel disease (IPG126)

Evidence-based recommendations on leukapheresis for inflammatory bowel disease (IBD) (ulcerative colitis and Crohn's disease)

Interventional procedures guidance Published June 2005

Endovascular stent–graft placement in thoracic aortic aneurysms and dissections (IPG127)

Evidence-based recommendations on endovascular stent–graft placement in thoracic aortic aneurysms (bulging aorta in the heart) and dissections (tears)

Interventional procedures guidance Published June 2005

Totally endoscopic robotically assisted coronary artery bypass grafting (IPG128)

Evidence-based recommendations on totally endoscopic robotically assisted coronary artery bypass grafting (TECAB)

Interventional procedures guidance Published June 2005

Computed tomographic colonography (virtual colonoscopy) (IPG129)

Evidence-based recommendations on computed tomographic (CT) colonography (virtual colonoscopy) to detect abnormalities in the colon

Interventional procedures guidance Published June 2005

Cryotherapy for recurrent prostate cancer (IPG119)

Evidence-based recommendations on cryotherapy for treating recurrent prostate cancer

Interventional procedures guidance Published May 2005

Percutaneous vertebroplasty (IPG12)

Evidence-based recommendations on percutaneous vertebroplasty for spinal metastases

Interventional procedures guidance Published September 2003

Radiofrequency ablation for atrial fibrillation in association with other cardiac surgery (IPG121)

Evidence-based recommendations on radiofrequency ablation for atrial fibrillation (AF) in association with other cardiac (heart) surgery

Interventional procedures guidance Published May 2005

Microwave ablation for atrial fibrillation in association with other cardiac surgery (IPG122)

Evidence-based recommendations on microwave ablation for atrial fibrillation (AF) in association with other cardiac (heart) surgery

Interventional procedures guidance Published May 2005

Cryoablation for atrial fibrillation in association with other cardiac surgery (IPG123)

Evidence-based recommendations on cryoablation for atrial fibrillation (AF) in association with other cardiac (heart) surgery

Interventional procedures guidance Published May 2005

Endoscopic dacryocystorhinostomy (IPG113)

Evidence-based recommendations on endoscopic dacryocystorhinostomy (DCR) for treating lacrimal sac or nasolacrimal duct obstruction (NLDO)

Interventional procedures guidance Published February 2005

Lung volume reduction surgery for advanced emphysema (IPG114)

Evidence-based recommendations on lung volume reduction surgery for treating advanced emphysema

Interventional procedures guidance Published February 2005

High-intensity focused ultrasound for prostate cancer (IPG118)

Evidence-based recommendations on high-intensity focused ultrasound for treating prostate cancer

Interventional procedures guidance Published March 2005

Coil embolisation of ruptured intracranial aneurysms (IPG106)

Evidence-based recommendations on coil embolisation for treating ruptured intracranial aneurysms

Interventional procedures guidance Published January 2005

Auditory brain stem implants (IPG108)

Evidence-based recommendations on auditory brain stem implants for deafness caused by damage to the vestibulocochlear nerve due to tumours or surgery

Interventional procedures guidance Published January 2005

Artificial metacarpophalangeal and interphalangeal joint replacement for end-stage arthritis (IPG110)

Evidence-based recommendations on artificial metacarpophalangeal and interphalangeal joint replacement for treating end-stage arthritis

Interventional procedures guidance Published February 2005

Artificial trapeziometacarpal joint replacement for end-stage osteoarthritis (IPG111)

Evidence-based recommendations on artificial trapeziometacarpal joint replacement for treating end-stage osteoarthritis

Interventional procedures guidance Published February 2005

Wireless capsule endoscopy for investigation of the small bowel (IPG101)

Evidence-based recommendations on wireless capsule endoscopy (swallowing small capsule with camera) for investigating the small bowel

Interventional procedures guidance Published December 2004

Coil embolisation of unruptured intracranial aneurysms (IPG105)

Evidence-based recommendations on coil embolisation (inserting coil) for treating unruptured intracranial aneurysms (dilated blood vessels in the skull)

Interventional procedures guidance Published January 2005

Collagen injection for vocal cord augmentation (IPG130)

Evidence-based recommendations collagen injection for vocal cord augmentation (in glottic insufficiency and vocal fold paresis)

Interventional procedures guidance Published June 2005

Interstitial laser therapy for fibroadenomas of the breast (IPG131)

Evidence-based recommendations on interstitial laser therapy for fibroadenomas (benign/non-cancerous solid lump) of the breast

Interventional procedures guidance Published June 2005

Cyanoacrylate instillation for occlusion of parotid sinuses (IPG42)

Evidence-based recommendations on cyanoacrylate instillation for occlusion of parotid sinuses

Interventional procedures guidance Published February 2004

Incisionless otoplasty (IPG422)

Evidence-based recommendations on incisionless otoplasty (surgery) to correct protruding ears

Interventional procedures guidance Published March 2012

Focal therapy using cryoablation for localised prostate cancer (IPG423)

Evidence-based recommendations on focal therapy using freezing (cryoablation) needles for treating localised prostate cancer

Interventional procedures guidance Published April 2012

Focal therapy using high-intensity focused ultrasound for localised prostate cancer (IPG424)

Evidence-based recommendations on focal therapy using high-intensity focused ultrasound for treating localised prostate cancer

Interventional procedures guidance Published April 2012

Endoscopic balloon dilatation for subglottic or tracheal stenosis (IPG425)

Evidence-based recommendations on endoscopic balloon dilatation for treating subglottic or tracheal stenosis (narrowing of the windpipe)

Interventional procedures guidance Published April 2012