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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

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

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

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

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

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

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

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

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

Human alpha1-proteinase inhibitor for treating emphysema ID856

In development [GID-HST10017] 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

Lysosomal acid lipase deficiency - sebelipase alfa [ID737]

In development [GID-LYSOSOMALACIDLIPASEDEFICIENCYSEBELIPASEALFAID737] Expected publication date: TBC

Highly specialised technologies guidance In development

Metreleptin for treating lipodystrophy (ID861)

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

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

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

Platelet-rich plasma injections for knee osteoarthritis (IPG637)

Evidence-based recommendations on platelet-rich plasma injections for knee osteoarthritis in adults (deterioration of the articular cartilage and menisci)

Interventional procedures guidance Published January 2019

Low-level laser therapy for preventing or treating oral mucositis caused by radiotherapy or chemotherapy (IPG615)

Evidence-based recommendations on low-level laser therapy for preventing or treating oral mucositis caused by radiotherapy or chemotherapy

Interventional procedures guidance Published May 2018

Artificial heart implantation as a bridge to transplantation for end-stage refractory biventricular heart failure (IPG602)

Evidence-based recommendations on artificial heart implantation as a bridge to transplantation for end-stage refractory biventricular heart failure

Interventional procedures guidance Published December 2017

Subcutaneous implantable cardioverter defibrillator insertion for preventing sudden cardiac death (IPG603)

Evidence-based recommendations on subcutaneous implantable cardioverter defibrillator insertion for preventing sudden cardiac death

Interventional procedures guidance Published December 2017

Transvaginal mesh repair of anterior or posterior vaginal wall prolapse (IPG599)

Evidence-based recommendations on transvaginal mesh repair of anterior or posterior vaginal wall prolapse

Interventional procedures guidance Published December 2017

Low-intensity pulsed ultrasound to promote healing of delayed-union and non-union fractures (IPG623)

Evidence-based recommendations on low-intensity pulsed ultrasound (LIPUS) to promote healing of delayed-union and non-union fractures

Interventional procedures guidance Published July 2018

Low-intensity pulsed ultrasound to promote healing of fresh fractures at high risk of non-healing (IPG622)

Evidence-based recommendations on low-intensity pulsed ultrasound (LIPUS) to promote healing of fresh fractures at high risk of non-healing

Interventional procedures guidance Published July 2018

Low-intensity pulsed ultrasound to promote healing of fresh fractures at low risk of non-healing (IPG621)

Evidence-based recommendations on low-intensity pulsed ultrasound (LIPUS) to promote healing of fresh fractures at low risk of non-healing in adults

Interventional procedures guidance Published July 2018

Transaxial interbody lumbosacral fusion for severe chronic low back pain (IPG620)

Evidence-based recommendations on transaxial interbody lumbosacral fusion for low back pain in adults

Interventional procedures guidance Published July 2018

Superior capsular augmentation for massive rotator cuff tears (IPG619)

Evidence-based recommendations on superior capsular augmentation for massive rotator cuff (shoulder) tears

Interventional procedures guidance Published July 2018

Laparoscopic mesh pectopexy for apical prolapse of the uterus or vagina (IPG608)

Evidence-based recommendations on laparoscopic mesh pectopexy for apical prolapse of the uterus or vagina

Interventional procedures guidance Published March 2018

Mosaicplasty for symptomatic articular cartilage defects of the knee (IPG607)

Evidence-based recommendations on mosaicplasty for symptomatic articular cartilage defects of the knee (osteochondral autologous transfer mosaicplasty)

Interventional procedures guidance Published March 2018

Unilateral MRI-guided focused ultrasound thalamotomy for moderate to severe tremor in Parkinson's disease (IPG606)

Evidence-based recommendations on unilateral MRI-guided focused ultrasound thalamotomy for moderate to severe tremor in Parkinson’s disease in adults

Interventional procedures guidance Published February 2018

Aortic valve reconstruction with processed bovine pericardium (IPG604)

Evidence-based recommendations on aortic valve reconstruction with processed bovine pericardium (using new valve made from chemically treated cow pericardium)

Interventional procedures guidance Published February 2018

Endobronchial valve insertion to reduce lung volume in emphysema (IPG600)

Evidence-based recommendations on endobronchial valve insertion to reduce lung volume in emphysema

Interventional procedures guidance Published December 2017

Transcutaneous microwave ablation for severe primary axillary hyperhidrosis (IPG601)

Evidence-based recommendations on transcutaneous microwave ablation for severe primary axillary hyperhidrosis (excessive sweating) in adults

Interventional procedures guidance Published December 2017

Sutureless aortic valve replacement for aortic stenosis (IPG624)

Evidence-based recommendations on sutureless aortic valve replacement for aortic stenosis in adults

Interventional procedures guidance Published August 2018

Superior rectal artery embolisation for haemorrhoids (IPG627)

Evidence-based recommendations on superior rectal artery embolisation for haemorrhoids in adults

Interventional procedures guidance Published August 2018

Leadless cardiac pacemaker implantation for bradyarrhythmias (IPG626)

Evidence-based recommendations on leadless cardiac pacemaker implantation for bradyarrhythmias (an inserted device to help the heart beat at a normal rate)

Interventional procedures guidance Published August 2018

Transurethral water jet ablation for lower urinary tract symptoms caused by benign prostatic hyperplasia (IPG629)

Evidence-based recommendations on transurethral water jet ablation for lower urinary tract symptoms (LUTS) caused by benign prostatic hyperplasia (BPH)

Interventional procedures guidance Published September 2018

Intravesical microwave hyperthermia and chemotherapy for non-muscle-invasive bladder cancer (IPG628)

Evidence-based recommendations on intravesical microwave hyperthermia and chemotherapy for non-muscle-invasive bladder cancer

Interventional procedures guidance Published September 2018

Direct C1 lateral mass screw for cervical spine stabilisation (IPG146)

Evidence-based recommendations on direct C1 lateral mass screw for cervical spine stabilisation (to treat atlantoaxial fusion in the neck)

Interventional procedures guidance Published December 2005

Photodynamic therapy for non-melanoma skin tumours (including premalignant and primary non-metastatic skin lesions) (IPG155)

Evidence-based recommendations on photodynamic therapy for non-melanoma skin tumours (including premalignant and primary non-metastatic skin lesions)

Interventional procedures guidance Published February 2006

Percutaneous radiofrequency ablation for atrial fibrillation (IPG168)

Evidence-based recommendations on percutaneous radiofrequency ablation for atrial fibrillation (AF)

Interventional procedures guidance Published April 2006

Percutaneous endoscopic colostomy (IPG161)

Evidence-based recommendations on percutaneous endoscopic colostomy (PEC)

Interventional procedures guidance Published March 2006