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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

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

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

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

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

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

Ravulizumab for treating paroxysmal nocturnal haemoglobinuria ID1457

Proposed [GID-HST10023] 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

Velmanase alfa for treating alpha-mannosidosis [ID800]

In development [GID-HST10010] 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

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

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

Intrathecal idursulfase for treating Mucopolysaccharidosis type II ID1223

Proposed [GID-HST10019] 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: 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

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]

In development [GID-HST10015] Expected publication date: 27 May 2020

Highly specialised technologies guidance In development

Improving outcomes in urological cancers (CSG2)

This guideline covers how healthcare services for people with urological cancer should be organised. It aims to improve care by recommending which healthcare professionals should be involved and the types of hospital or cancer centre best suited to provide the care.

Cancer service guideline Published September 2002

Improving outcomes for people with sarcoma (CSG9)

This guideline covers how healthcare services for people with sarcoma should be organised. It aims to improve care by recommending which healthcare professionals should be involved and the types of hospital or cancer centre best suited to provide the care.

Cancer service guideline Published March 2006

Improving outcomes for people with skin tumours including melanoma (CSG8)

This guideline covers how healthcare services for people with skin cancers should be organised. It aims to improve diagnosis and care by recommending which healthcare professionals should be involved and at which stage.

Cancer service guideline Published February 2006 Last updated May 2010

Improving outcomes in head and neck cancers (CSG6)

This guideline covers how healthcare services for adults with head and neck cancers should be organised. It aims to improve care by recommending which healthcare professionals should be involved and the types of hospital or cancer centre best suited to provide the care.

Cancer service guideline Published November 2004

Improving outcomes in children and young people with cancer (CSG7)

This guideline covers how healthcare services for children and young people with cancer should be organised. It aims to improve care by recommending which healthcare professionals should be involved and the types of hospital or cancer centre best suited to provide the care.

Cancer service guideline Published August 2005

Improving supportive and palliative care for adults with cancer (CSG4)

This guideline covers best practice in developing and delivering cancer services for adults. It aims to ensure that people with cancer, and their families and carers, are well informed, cared for and supported from before formal diagnosis onward.

Cancer service guideline Published March 2004

Improving outcomes in breast cancer (CSG1)

This guideline covers how healthcare services for breast cancer should be organised. It aims to improve care for women with breast cancer by recommending which healthcare professionals should be involved in care.

Cancer service guideline Published August 2002

Improving outcomes for people with brain and other central nervous system tumours (CSG10)

This guideline covers how healthcare services for people with brain tumours and other central nervous system tumours should be organised. It aims to improve care by recommending which healthcare professionals should be involved and the types of hospital or cancer centre best suited to provide the care.

Cancer service guideline Published June 2006

Ulcerative colitis: management (NG130)

This guideline covers the management of ulcerative colitis in children, young people and adults. It aims to help professionals to provide consistent high-quality care and it highlights the importance of advice and support for people with ulcerative colitis.

NICE guideline Published May 2019

Prostate cancer: diagnosis and management (NG131)

This guideline covers the diagnosis and management of prostate cancer in secondary care, including information on the best way to diagnose and identify different stages of the disease, and how to manage adverse effects of treatment. It also includes recommendations on follow-up in primary care for people diagnosed with prostate cancer.

NICE guideline Published May 2019

Suspected neurological conditions: recognition and referral (NG127)

This guideline covers the initial assessment of symptoms and signs that might indicate a neurological condition. It helps non-specialist healthcare professionals to identify people who should be offered referral for specialist investigation.

NICE guideline Published May 2019 Last updated July 2019

Stroke and transient ischaemic attack in over 16s: diagnosis and initial management (NG128)

This guideline covers interventions in the acute stage of a stroke or transient ischaemic attack (TIA). It offers the best clinical advice on the diagnosis and acute management of stroke and TIA in the 48 hours after onset of symptoms.

NICE guideline Published May 2019

Crohn's disease: management (NG129)

This guideline covers the management of Crohn’s disease in children, young people and adults. It aims to reduce people’s symptoms and maintain or improve their quality of life.

NICE guideline Published May 2019

Hypertension in pregnancy: diagnosis and management (NG133)

This guideline covers diagnosing and managing hypertension (high blood pressure), including pre-eclampsia, during pregnancy, labour and birth. It also includes advice for women with hypertension who wish to conceive and women who have had a pregnancy complicated by hypertension. It aims to improve care during pregnancy, labour and birth for women and their babies.

NICE guideline Published June 2019

Melanoma: assessment and management (NG14)

This guideline covers the assessment and management of melanoma (a type of skin cancer) in children, young people and adults. It aims to reduce variation in practice and improve survival.

NICE guideline Published July 2015

Surgical site infections: prevention and treatment (NG125)

This guideline covers preventing and treating surgical site infections in adults, young people and children who are having a surgical procedure involving a cut through the skin. It focuses on methods used before, during and after surgery to minimise the risk of infection.

NICE guideline Published April 2019

Ectopic pregnancy and miscarriage: diagnosis and initial management (NG126)

This guideline covers diagnosing and managing ectopic pregnancy and miscarriage in women with complications, such as pain and bleeding, in early pregnancy (that is, up to 13 completed weeks of pregnancy). It aims to improve how early pregnancy loss is diagnosed, and the support women are given, to limit the psychological impact of their loss.

NICE guideline Published April 2019

Depression in children and young people: identification and management (NG134)

This guideline covers identifying and managing depression in children and young people aged 5 to 18 years. Based on the stepped-care model, it aims to improve recognition and assessment and promote effective treatments for mild and moderate to severe depression.

NICE guideline Published June 2019

Specialist neonatal respiratory care for babies born preterm (NG124)

This guideline covers specific aspects of respiratory support (for example, oxygen supplementation, assisted ventilation, treatment of some respiratory disorders, and aspects of monitoring) for preterm babies in hospital.

NICE guideline Published April 2019

Depression in adults: recognition and management (CG90)

This guideline covers identifying and managing depression in adults aged 18 years and older, in primary and secondary care. It aims to improve care for people with depression by promoting improved recognition and treatment.

Clinical guideline Published October 2009

Depression in adults with a chronic physical health problem: recognition and management (CG91)

This guideline covers identifying, treating and managing depression in people aged 18 and over who also have a chronic physical health problem such as cancer, heart disease or diabetes. It aims to improve the care of people with a long-term physical health problem, which can cause or exacerbate depression. This has the potential to increase their quality of life and life expectancy.

Clinical guideline Published October 2009

Donor milk banks: service operation (CG93)

This guideline covers how donor milk banks should recruit, screen and support women who donate breast milk. It also covers how milk banks should handle and process the breast milk they receive from donors. It aims to improve the safety of donor milk and operation of donor milk services.

Clinical guideline Published February 2010

Unstable angina and NSTEMI: early management (CG94)

This guideline covers treatments for people aged 18 and over with unstable angina (recurring chest pain) or a type of heart attack called non-ST-segment-elevation myocardial infarction (NSTEMI). It aims to ensure that people get treatment quickly. It recommends that as soon as NSTEMI or unstable angina is diagnosed, healthcare professionals assess people for risk of more serious heart problems in the future to guide their treatment.

Clinical guideline Published March 2010 Last updated November 2013

Recent-onset chest pain of suspected cardiac origin: assessment and diagnosis (CG95)

This guideline covers assessing and diagnosing recent chest pain in people aged 18 and over and managing symptoms while a diagnosis is being made. It aims to improve outcomes by providing advice on tests (ECG, high-sensitivity troponin tests, multislice CT angiography, functional testing) that support healthcare professionals to make a speedy and accurate diagnosis.

Clinical guideline Published March 2010 Last updated November 2016

Lower urinary tract symptoms in men: management (CG97)

This guideline covers managing lower urinary tract symptoms (LUTS) in men over 18. It aims to improve the quality of life for men with LUTS by recommending which assessments they should receive, and when conservative management, drug treatment and surgery can help.

Clinical guideline Published May 2010 Last updated June 2015