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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

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

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

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

Volanesorsen for treating familial chylomicronaemia syndrome [ID1326]

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

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

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

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

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

Cardiovascular disease prevention (PH25)

This guideline covers the main risk factors linked with cardiovascular disease: poor diet, physical inactivity, smoking and excessive alcohol consumption. It aims to reduce the high incidence of cardiovascular disease. This, in turn, will help prevent other major causes of death and illness, such as type 2 diabetes and many cancers.

Public health guideline Published June 2010

Smoking prevention in schools (PH23)

This guideline covers smoking prevention interventions that are delivered in schools or other educational institutions. It aims to prevent children and young people aged under 19 from taking up smoking.

Public health guideline Published February 2010

Alcohol-use disorders: prevention (PH24)

This guideline covers alcohol problems among people over 10. It aims to prevent and identify such problems as early as possible using a mix of policy and practice.

Public health guideline Published June 2010

Looked-after children and young people (PH28)

This guideline covers how organisations, professionals and carers can work together to deliver high quality care, stable placements and nurturing relationships for looked-after children and young people. It aims to help these children and young people reach their full potential and enjoy the same opportunities in life as their peers.

Public health guideline Published October 2010 Last updated May 2015

Unintentional injuries: prevention strategies for under 15s (PH29)

This guideline covers strategies, regulation, enforcement, surveillance and workforce development in relation to preventing unintentional injuries in the home, on the road and during outdoor play and leisure.

Public health guideline Published November 2010

Social and emotional wellbeing in secondary education (PH20)

This guideline covers interventions to support social and emotional wellbeing among young people aged 11–19 years who are in full-time education. It aims to promote good social, emotional and psychological health to protect young people against behavioural and health problems.

Public health guideline Published September 2009

Immunisations: reducing differences in uptake in under 19s (PH21)

This guideline covers increasing immunisation uptake among children and young people aged under 19 years in groups and settings where immunisation coverage is low. It aims to improve access to immunisation services and increase timely immunisation of children and young people. It also aims to ensure babies born to mothers infected with hepatitis B are immunised.

Public health guideline Published September 2009 Last updated September 2017

Sexually transmitted infections and under-18 conceptions: prevention (PH3)

This guideline covers one-to-one interventions to prevent sexually transmitted infections (STIs) and under-18 conceptions. The aim is to reduce the transmission of chlamydia and other STIs, including HIV, and reduce the rate of pregnancies among women aged under 18.

Public health guideline Published February 2007

Unintentional injuries in the home: interventions for under 15s (PH30)

This guideline covers home safety assessments, supplying and installing safety equipment and providing education and advice. It aims to prevent unintentional injuries among all children and young people aged under 15 but, in particular, those living in disadvantaged circumstances.

Public health guideline Published November 2010

Unintentional injuries on the road: interventions for under 15s (PH31)

This guideline covers road speed limits, 20mph zones and engineering measures to reduce speed or make routes safer.

Public health guideline Published November 2010

Skin cancer prevention (PH32)

This guideline covers new buildings and communal outdoor areas. The aim is to ensure there are enough shady areas to protect people from overexposure to the sun.

Public health guideline Published January 2011 Last updated February 2016

Weight management before, during and after pregnancy (PH27)

This guideline covers how to assess and monitor body weight and how to prevent someone from becoming overweight or obese before, during and after pregnancy. The aim is help all women who have a baby to achieve and maintain a healthy weight by adopting a balanced diet and being physically active.

Public health guideline Published July 2010

Smoking: stopping in pregnancy and after childbirth (PH26)

This guideline covers support to help women stop smoking during pregnancy and in the first year after childbirth. It includes identifying women who need help to quit, referring them to stop smoking services and providing intensive and ongoing support to help them stop. The guideline also advises how to tailor services for women from disadvantaged groups in which smoking rates are high.

Public health guideline Published June 2010

Mental wellbeing at work (PH22)

This guideline covers how to create the right conditions to support mental wellbeing at work. The aim is to promote a culture of participation, equality and fairness in the workplace based on open communication and flexible working.

Public health guideline Published November 2009

Intrapartum care for healthy women and babies (CG190)

This guideline covers the care of healthy women and their babies, during labour and immediately after the birth. It focuses on women who give birth between 37 and 42 weeks of pregnancy (‘term’). The guideline helps women to make an informed choice about where to have their baby. It also aims to reduce variation in areas of care such as fetal monitoring during labour and management of the third stage of labour.

Clinical guideline Published December 2014 Last updated February 2017

Pneumonia in adults: diagnosis and management (CG191)

This guideline covers diagnosing and managing community- and hospital-acquired pneumonia in adults. It aims to improve accurate assessment and diagnosis of pneumonia to help guide antibiotic prescribing and ensure that people receive the right treatment.

Clinical guideline Published December 2014 Last updated September 2019

Long-acting reversible contraception (CG30)

This guideline covers long-acting reversible contraception. It aims to increase the use of long-action reversible contraception by improving the information given to women about their contraceptive choices.

Clinical guideline Published October 2005 Last updated July 2019