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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

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

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

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

Velmanase alfa for treating alpha-mannosidosis [ID800]

In development [GID-HST10010] 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: 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

Intrathecal idursulfase for treating Mucopolysaccharidosis type II ID1223

Proposed [GID-HST10019] Expected publication date: TBC

Highly specialised technologies guidance Proposed

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

Proposed [GID-HST10020] 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

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

OTL-200 for treating metachromatic leukodystrophy [ID1666]

Proposed [GID-HST10028] 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

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

Meningitis (bacterial) and meningococcal septicaemia in under 16s: recognition, diagnosis and management (CG102)

This guideline covers recognising, diagnosing and managing bacterial meningitis and meningococcal septicaemia (blood poisoning) in babies, children and young people under 16. It aims to reduce deaths and disability by promoting early recognition of symptoms and timely effective management.

Clinical guideline Published June 2010 Last updated February 2015

Delirium: prevention, diagnosis and management (CG103)

This guideline covers diagnosing and treating delirium in people aged 18 and over in hospital and in long-term residential care or a nursing home. It also covers identifying people at risk of developing delirium in these settings and preventing onset. It aims to improve diagnosis of delirium and reduce hospital stays and complications.

Clinical guideline Published July 2010 Last updated March 2019

Metastatic malignant disease of unknown primary origin in adults: diagnosis and management (CG104)

This guideline covers diagnosing and managing secondary cancers in people aged 18 and over when the site of the primary cancer is unknown. This includes people who have had treatment for cancer before. It aims to improve quality of life by offering advice on tests for identifying the site of the primary cancer and options for managing the person’s condition when this cannot be found.

Clinical guideline Published July 2010

Sedation in under 19s: using sedation for diagnostic and therapeutic procedures (CG112)

This guideline covers the assessment, preparation, training and monitoring needed when using sedation in people aged under 19. It aims to help healthcare professionals decide when sedation is the most clinically and cost effective option for reducing pain and anxiety during operations for children and young people.

Clinical guideline Published December 2010

Generalised anxiety disorder and panic disorder in adults: management (CG113)

This guideline covers the care and treatment of people aged 18 and over with generalised anxiety disorder (chronic anxiety) or panic disorder (with or without agoraphobia or panic attacks). It aims to help people achieve complete relief of symptoms (remission), which is associated with better functioning and a lower likelihood of relapse.

Clinical guideline Published January 2011 Last updated July 2019

Alcohol-use disorders: diagnosis, assessment and management of harmful drinking (high-risk drinking) and alcohol dependence (CG115)

This guideline covers identifying, assessing and managing alcohol-use disorders (harmful drinking and alcohol dependence) in adults and young people aged 10–17 years. It aims to reduce harms (such as liver disease, heart problems, depression and anxiety) from alcohol by improving assessment and setting goals for reducing alcohol consumption.

Clinical guideline Published February 2011

Colorectal cancer (NG151)

This guideline covers managing colorectal (bowel) cancer in people aged 18 and over. It aims to improve quality of life and survival for adults with colorectal cancer through management of local disease and management of secondary tumours (metastatic disease).

NICE guideline Published January 2020

Head injury: assessment and early management (CG176)

This guideline covers the assessment and early management of head injury in children, young people and adults. It promotes effective clinical assessment so that people receive the right care for the severity of their head injury, including referral directly to specialist care if needed.

Clinical guideline Published January 2014 Last updated September 2019

Osteoarthritis: care and management (CG177)

This guideline covers assessing and managing osteoarthritis in adults. It covers both pharmacological and non-pharmacological treatments. It promotes effective treatment options to control joint pain and improve function in people with osteoarthritis.

Clinical guideline Published February 2014

Psychosis and schizophrenia in adults: prevention and management (CG178)

This guideline covers recognising and managing psychosis and schizophrenia in adults. It aims to improve care through early recognition and treatment, and by focusing on long-term recovery. It also recommends checking for coexisting health problems and providing support for family members and carers.

Clinical guideline Published February 2014 Last updated March 2014

Social anxiety disorder: recognition, assessment and treatment (CG159)

This guideline covers recognising, assessing and treating social anxiety disorder (also known as ‘social phobia’) in children and young people (from school age to 17 years) and adults (aged 18 years and older). It aims to improve symptoms, educational, occupational and social functioning, and quality of life in people with social anxiety disorder.

Clinical guideline Published May 2013

Self-harm in over 8s: short-term management and prevention of recurrence (CG16)

This guideline covers the short-term management and prevention of self-harm in people aged 8 and over, regardless of whether accompanied by mental illness. It covers the first 48 hours following an act of self-harm, but does not address the longer-term psychiatric care of people who self-harm.

Clinical guideline Published July 2004

Falls in older people: assessing risk and prevention (CG161)

This guideline covers assessment of fall risk and interventions to prevent falls in people aged 65 and over. It aims to reduce the risk and incidence of falls and the associated distress, pain, injury, loss of confidence, loss of independence and mortality.

Clinical guideline Published June 2013

Stroke rehabilitation in adults (CG162)

This guideline covers stroke rehabilitation for adults and young people aged 16 and over who have had a stroke with continuing impairment, activity limitation or participation restriction. It aims to improve rehabilitation for people who have had a stroke by specifying how stroke units and multidisciplinary stroke teams should be organised. It makes detailed recommendations on assessments and interventions for the functional difficulties caused by stroke.

Clinical guideline Published June 2013

Idiopathic pulmonary fibrosis in adults: diagnosis and management (CG163)

This guideline covers diagnosing and managing idiopathic pulmonary fibrosis in people aged 18 and over. It aims to improve the quality of life for people with idiopathic pulmonary fibrosis by helping healthcare professionals to diagnose the condition and provide effective symptom management.

Clinical guideline Published June 2013 Last updated May 2017

Psoriasis: assessment and management (CG153)

This guideline covers assessing and managing psoriasis in adults, young people and children. It aims to improve long-term disease control and quality of life for people with psoriasis.

Clinical guideline Published October 2012 Last updated September 2017

Chronic kidney disease (stage 4 or 5): management of hyperphosphataemia (CG157)

This guideline covers managing hyperphosphataemia in children, young people and adults with stage 4 or 5 chronic kidney disease. It makes recommendations on dietary management and phosphate binders, to reduce variation in care and the risk of hyperparathyroidism for people with chronic kidney disease.

Clinical guideline Published March 2013

Antisocial behaviour and conduct disorders in children and young people: recognition and management (CG158)

This guideline covers recognising and managing antisocial behaviour and conduct disorders in children and young people aged under 19. It aims to improve care by identifying children and young people who are at risk and when interventions can prevent conduct disorders from developing. The guideline also makes recommendations on communication, to help professionals build relationships with children and young people and involve them in their own care.

Clinical guideline Published March 2013 Last updated April 2017

Myocardial infarction with ST-segment elevation: acute management (CG167)

This guideline covers care and treatment of people aged 18 and over with a type of heart attack known as spontaneous onset of myocardial infarction with ST-segment elevation (STEMI). It aims to ensure that adults with STEMI are assessed and treated as soon as possible to minimise the damage to their heart. It also aims to help commissioners and healthcare professionals configure services so that people with STEMI can have the best outcomes.

Clinical guideline Published July 2013

Varicose veins: diagnosis and management (CG168)

This guideline covers diagnosing and managing varicose veins in people aged 18 and over. It aims to ensure that people understand the options for treating varicose veins and that healthcare professionals know when to refer people for specialist assessment and treatment.

Clinical guideline Published July 2013

Autism spectrum disorder in under 19s: support and management (CG170)

This guideline covers children and young people with autism spectrum disorder (across the full range of intellectual ability) from birth until their 19th birthday. It covers the different ways that health and social care professionals can provide support, treatment and help for children and young people with autism, and their families and carers, from the early years through to their transition into young adult life.

Clinical guideline Published August 2013

Fertility problems: assessment and treatment (CG156)

This guideline covers diagnosing and treating fertility problems. It aims to reduce variation in practice and improve the way fertility problems are investigated and managed.

Clinical guideline Published February 2013 Last updated September 2017

Myocardial infarction: cardiac rehabilitation and prevention of further cardiovascular disease (CG172)

This guideline covers cardiac rehabilitation and preventing further cardiovascular disease in people aged 18 and over who have had a myocardial infarction. It aims to promote the health of people who have had an MI by encouraging them to attend a cardiac rehabilitation programme and advising them on a healthy lifestyle. It also includes advice on drug therapy.

Clinical guideline Published November 2013

Hepatitis B (chronic): diagnosis and management (CG165)

This guideline covers assessing and managing chronic hepatitis B in children, young people and adults. It aims to improve care for people with hepatitis B by specifying which tests and treatments to use for people of different ages and with different disease severities.

Clinical guideline Published June 2013 Last updated October 2017