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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

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

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

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

Highly specialised technologies guidance Published February 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

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

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

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

Intrathecal idursulfase for treating Mucopolysaccharidosis type II ID1223

Proposed [GID-HST10019] Expected publication date: TBC

Highly specialised technologies guidance Proposed

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

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

Metreleptin for treating lipodystrophy (ID861)

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

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

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-200 for treating metachromatic leukodystrophy [ID1666]

Proposed [GID-HST10028] 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

Velmanase alfa for treating alpha-mannosidosis [ID800]

In development [GID-HST10010] Expected publication date: TBC

Highly specialised technologies guidance In development

Service user experience in adult mental health: improving the experience of care for people using adult NHS mental health services (CG136)

This guideline covers the components of a good experience of service use. It aims to make sure that all adults using NHS mental health services have the best possible experience of care.

Clinical guideline Published December 2011

Bronchiolitis in children: diagnosis and management (NG9)

This guideline covers diagnosing and managing bronchiolitis in children. It aims to help healthcare professionals diagnose bronchiolitis and identify if children should be cared for at home or in hospital. It describes treatments and interventions that can be used to help with the symptoms of bronchiolitis.

NICE guideline Published June 2015

Chronic kidney disease: managing anaemia (NG8)

This guideline covers diagnosing, assessing, managing and monitoring anaemia in people with chronic kidney disease. It aims to improve care for people with chronic kidney disease by specifying how to assess when their anaemia needs treating, and by making detailed recommendations on treatment with erythropoietic stimulating agents (ESAs) and iron. It also covers detecting and managing ESA-resistant anaemia.

NICE guideline Published June 2015

Urinary incontinence in neurological disease: assessment and management (CG148)

This guideline covers assessing and managing urinary incontinence in children, young people and adults with neurological disease. It aims to improve care by recommending specific treatments based on what symptoms and neurological conditions people have.

Clinical guideline Published August 2012

Healthcare-associated infections: prevention and control in primary and community care (CG139)

This guideline covers preventing and controlling healthcare-associated infections in children, young people and adults in primary and community care settings. It provides a blueprint for the infection prevention and control precautions that should be applied by everyone involved in delivering NHS care and treatment.

Clinical guideline Published March 2012 Last updated February 2017

Palliative care for adults: strong opioids for pain relief (CG140)

This guideline covers safe and effective prescribing of strong opioids for pain relief in adults with advanced and progressive disease. It aims to clarify the clinical pathway for prescribing and help to improve pain management and patient safety. Care during the last 2 to 3 days of life is covered by care of dying adults in the last days of life.

Clinical guideline Published May 2012 Last updated August 2016

Acute upper gastrointestinal bleeding in over 16s: management (CG141)

This guideline covers how upper gastrointestinal bleeding can be effectively managed in adults and young people aged 16 years and older. It aims to identify which diagnostic and therapeutic steps are useful so hospitals can develop a structure in which clinical teams can deliver an optimum service for people who develop this condition.

Clinical guideline Published June 2012 Last updated August 2016

Autism spectrum disorder in adults: diagnosis and management (CG142)

This guideline covers diagnosing and managing suspected or confirmed autism spectrum disorder (autism, Asperger’s syndrome and atypical autism) in people aged 18 and over. It aims to improve access and engagement with interventions and services, and the experience of care, for people with autism.

Clinical guideline Published June 2012 Last updated August 2016

Sickle cell disease: managing acute painful episodes in hospital (CG143)

This guideline covers managing acute painful sickle cell episodes in children, young people and adults who present at hospital, from presentation until when they are discharged. It aims to reduce variation in how acute episodes are managed in hospital, focusing on effective, prompt and safe pain relief.

Clinical guideline Published June 2012

Venous thromboembolic diseases: diagnosis, management and thrombophilia testing (CG144)

This guideline covers the diagnosis and management of venous thromboembolic diseases in adults (aged 18 and over), and the role of thrombophilia testing. It aims to clarify which diagnostic tests should be used, reduce variation in pharmacological therapies, and provide guidance on when thrombophilia testing is useful.

Clinical guideline Published June 2012 Last updated November 2015

Self-harm in over 8s: long-term management (CG133)

This guideline covers the longer-term psychological treatment and management of self-harm in people aged 8 and over. It aims to improve the quality of care and support for people who self harm and covers both single and recurrent episodes of self-harm.

Clinical guideline Published November 2011

Anaphylaxis: assessment and referral after emergency treatment (CG134)

This guideline covers assessment and referral for anaphylaxis. It aims to improve the quality of care for people with suspected anaphylaxis by detailing the assessments that are needed and recommending referral to specialist allergy services.

Clinical guideline Published December 2011

Organ donation for transplantation: improving donor identification and consent rates for deceased organ donation (CG135)

This guideline covers identifying people who wish to donate their organs after their death. It offers advice on how to approach families and carers of people who are nearing the end of life and how to seek consent for organ donation. It aims to promote discussion of organ donation as part of end-of-life care and to increase the number of organs available for people waiting for a transplant.

Clinical guideline Published December 2011 Last updated December 2016

Neonatal infection (early onset): antibiotics for prevention and treatment (CG149)

This guideline covers preventing infection within 72 hours of birth in healthy babies, treating pregnant women whose baby is at risk, and caring for babies who have a suspected or confirmed infection. It aims to reduce delays in recognising and treating sick babies and prevent unnecessary use of antibiotics.

Clinical guideline Published August 2012

Headaches in over 12s: diagnosis and management (CG150)

This guideline covers advice on the diagnosis and management of tension-type headache, migraine (including migraine with aura and menstrual-related migraine), cluster headache and medication overuse headache in young people (aged 12 years and older) and adults. It aims to improve the recognition and management of headaches, with more targeted treatment to improve the quality of life for people with headaches, and to reduce unnecessary investigations.

Clinical guideline Published September 2012 Last updated November 2015

Neutropenic sepsis: prevention and management in people with cancer (CG151)

This guideline covers preventing, identifying and managing neutropenic sepsis in children, young people and adults receiving treatment for cancer in the community and in secondary and tertiary care. It aims to reduce the risk of infection in people with neutropenia (low number of white blood cells) who are receiving anticancer treatment and improve management of neutropenic sepsis.

Clinical guideline Published September 2012

Coexisting severe mental illness (psychosis) and substance misuse: assessment and management in healthcare settings (CG120)

This guideline covers assessing and managing people aged 14 years and over with coexisting severe mental illness (psychosis) and substance misuse. It aims to help healthcare professionals guide people with psychosis with coexisting substance misuse to stabilise, reduce or stop their substance misuse, to improve treatment adherence and outcomes, and to enhance their lives.

Clinical guideline Published March 2011

Ovarian cancer: recognition and initial management (CG122)

This guideline covers detecting, diagnosing and treating women (18 years and older) who have, or are suspected of having, epithelial ovarian cancer, fallopian tube cancer, primary peritoneal cancer or borderline ovarian cancer. It aims to enable earlier detection of ovarian cancer and improve initial treatment.

Clinical guideline Published April 2011

Hyperglycaemia in acute coronary syndromes: management (CG130)

This guideline covers managing hyperglycaemia in the first 48 hours for adults admitted to hospital for acute coronary syndromes. It aims to improve initial management of hyperglycaemia and ensure that adults are given advice on their diabetes risk and how to reduce it.

Clinical guideline Published October 2011

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

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

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

Suspected cancer: recognition and referral (NG12)

This guideline covers identifying children, young people and adults with symptoms that could be caused by cancer. It outlines appropriate investigations in primary care, and selection of people to refer for a specialist opinion. It aims to help people understand what to expect if they have symptoms that may suggest cancer.

NICE guideline Published June 2015 Last updated July 2017

Intravenous fluid therapy in children and young people in hospital (NG29)

This guideline covers general principles for managing intravenous (IV) fluids for children and young people under 16 years, including assessing fluid and electrolyte status and prescribing IV fluid therapy. It applies to a range of conditions and different settings. It does not include recommendations relating to specific conditions. This guideline represents a major opportunity to improve patient safety for children and young people having IV fluid therapy in hospital.

NICE guideline Published December 2015

Diabetes in pregnancy: management from preconception to the postnatal period (NG3)

This guideline covers managing diabetes and its complications in women who are planning pregnancy or are already pregnant. It aims to improve the diagnosis of gestational diabetes and help women with diabetes to self-manage their blood glucose levels before and during pregnancy.

NICE guideline Published February 2015 Last updated August 2015