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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

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

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 (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

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

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

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

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

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

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

Proposed [GID-HST10020] 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

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

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

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

Human alpha1-proteinase inhibitor for treating emphysema ID856

In development [GID-HST10017] 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

Metreleptin for treating lipodystrophy (ID861)

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

Highly specialised technologies guidance In development

Volanesorsen for treating familial chylomicronaemia syndrome [ID1326]: evaluation consultation

We are listening to your views on this highly specialised technology. Comments close 27 January 2020.

Highly specialised technologies guidance In consultation

Therapeutic hypothermia with intracorporeal temperature monitoring for hypoxic perinatal brain injury (IPG347)

Evidence-based recommendations on therapeutic hypothermia with intracorporeal temperature monitoring for treating hypoxic perinatal brain injury

Interventional procedures guidance Published May 2010

Mini-incision surgery for total knee replacement (IPG345)

Evidence-based recommendations on mini-incision surgery for total knee replacement

Interventional procedures guidance Published May 2010

Epithelial radiofrequency ablation for Barrett's oesophagus (IPG344)

Evidence-based recommendations on epithelial radiofrequency ablation for treating Barrett's oesophagus

Interventional procedures guidance Published May 2010

Haemorrhoidal artery ligation (IPG342)

Evidence-based recommendations on haemorrhoidal artery ligation for treating haemorrhoids (piles)

Interventional procedures guidance Published May 2010

Prosthetic intervertebral disc replacement in the cervical spine (IPG341)

Evidence-based recommendations on prosthetic intervertebral disc replacement in the cervical spine for treating degenerative cervical disc disease

Interventional procedures guidance Published May 2010

Macular translocation with 360° retinotomy for wet age-related macular degeneration (IPG340)

Evidence-based recommendations on macular translocation with 360° retinotomy for treating wet (exudative) age-related macular degeneration (AMD)

Interventional procedures guidance Published May 2010

Circular stapled haemorrhoidectomy (IPG34)

Evidence-based recommendations on circular stapled haemorrhoidectomy for treating internal haemorrhoids

Interventional procedures guidance Published December 2003

Limited macular translocation for wet age-related macular degeneration (IPG339)

Evidence-based recommendations on limited macular translocation for treating wet (exudative) age-related macular degeneration (AMD)

Interventional procedures guidance Published May 2010

Laparoscopic radical hysterectomy for early stage cervical cancer (IPG338)

Evidence-based recommendations on laparoscopic radical hysterectomy for treating early stage cervical cancer

Interventional procedures guidance Published May 2010

Endobronchial ultrasound-guided transbronchial biopsy for peripheral lung lesions (IPG337)

Evidence-based recommendations on endobronchial ultrasound-guided transbronchial biopsy for treating peripheral lung lesions (pulmonary nodules)

Interventional procedures guidance Published March 2010

Transcatheter endovascular closure of perimembranous ventricular septal defect (IPG336)

Evidence-based recommendations on transcatheter endovascular closure of perimembranous ventricular septal defect (VSD) for treating holes in the heart

Interventional procedures guidance Published March 2010

Endoscopic submucosal dissection of lower gastrointestinal lesions (IPG335)

Evidence-based recommendations on endoscopic submucosal dissection of lower gastrointestinal lesions

Interventional procedures guidance Published March 2010

Arteriovenous crossing sheathotomy for branch retinal vein occlusion (IPG334)

Evidence-based recommendations on arteriovenous crossing sheathotomy for treating branch retinal vein occlusion (BRVO, retinal vascular thrombosis)

Interventional procedures guidance Published March 2010

Therapeutic endoscopic division of epidural adhesions (IPG333)

Evidence-based recommendations on therapeutic endoscopic division of epidural adhesions for back or leg pain from adhesions around the spinal nerve roots

Interventional procedures guidance Published February 2010

Surgical correction of hallux valgus using minimal access techniques (IPG332)

Evidence-based recommendations on surgical correction using minimal access techniques for treating hallux valgus (bunions)

Interventional procedures guidance Published February 2010

Cytoreduction surgery followed by hyperthermic intraoperative peritoneal chemotherapy for peritoneal carcinomatosis (IPG331)

Evidence-based recommendations on cytoreduction surgery and hyperthermic intraoperative peritoneal chemotherapy for peritoneal carcinomatosis (cancer)

Interventional procedures guidance Published February 2010

Vagus nerve stimulation for treatment-resistant depression (IPG330)

Evidence-based recommendations on vagus nerve stimulation for treatment-resistant depression

Interventional procedures guidance Published December 2009

Endoaortic balloon occlusion for cardiac surgery (IPG261)

Evidence-based recommendations on endoaortic balloon occlusion for heart surgery

Interventional procedures guidance Published May 2008

Interstitial photodynamic therapy for malignant parotid tumours (IPG259)

Evidence-based recommendations on interstitial photodynamic therapy (PDT) for treating malignant parotid tumours

Interventional procedures guidance Published April 2008

Intraoperative red blood cell salvage during radical prostatectomy or radical cystectomy (IPG258)

Evidence-based recommendations on intraoperative red blood cell salvage during radical surgery to remove the prostate gland or bladder

Interventional procedures guidance Published April 2008

Allogeneic pancreatic islet cell transplantation for type 1 diabetes mellitus (IPG257)

Evidence-based recommendations on allogeneic pancreatic islet cell transplantation for treating type 1 diabetes mellitus

Interventional procedures guidance Published April 2008

Intraoperative nerve monitoring during thyroid surgery (IPG255)

Evidence-based recommendations on intraoperative nerve monitoring during thyroid surgery

Interventional procedures guidance Published March 2008

Endobronchial ultrasound-guided transbronchial needle aspiration for mediastinal masses (IPG254)

Evidence-based recommendations on endobronchial ultrasound-guided transbronchial needle aspiration (EBUS-TBNA) for mediastinal masses

Interventional procedures guidance Published February 2008

Laparoscopic mobilisation of the greater omentum for breast reconstruction (IPG253)

Evidence-based recommendations on laparoscopic mobilisation of the greater omentum (keyhole surgery) for breast reconstruction

Interventional procedures guidance Published February 2008