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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

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

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

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

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

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

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

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

Metreleptin for treating lipodystrophy (ID861)

In development [GID-HST10011] 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

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

Proposed [GID-HST10020] 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

Intrathecal idursulfase for treating Mucopolysaccharidosis type II ID1223

Proposed [GID-HST10019] 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

Afamelanotide for treating erythropoietic protoporphyria [ID927]

In development [GID-HST10009] 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

Velmanase alfa for treating alpha-mannosidosis [ID800]

In development [GID-HST10010] 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

Human alpha1-proteinase inhibitor for treating emphysema ID856

In development [GID-HST10017] Expected publication date: TBC

Highly specialised technologies guidance In development

Cerliponase alfa for treating neuronal ceroid lipofuscinosis type 2 [ID943]

In development [GID-HST10008] Expected publication date: 27 November 2019

Highly specialised technologies guidance In development

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

OTL-200 for treating metachromatic leukodystrophy [ID1666]

Proposed [GID-HST10028] Expected publication date: TBC

Highly specialised technologies guidance Proposed

Diabetes (type 1 and type 2) in children and young people: diagnosis and management (NG18)

This guideline covers the diagnosis and management of type 1 and type 2 diabetes in children and young people aged under 18. The guideline recommends strict targets for blood glucose control to reduce the long-term risks associated with diabetes.

NICE guideline Published August 2015 Last updated November 2016

Children's attachment: attachment in children and young people who are adopted from care, in care or at high risk of going into care (NG26)

This guideline covers the identification, assessment and treatment of attachment difficulties in children and young people up to age 18 who are adopted from care, in special guardianship, looked after by local authorities in foster homes (including kinship foster care), residential settings and other accommodation, or on the edge of care. It aims to address the many emotional and psychological needs of children and young people in these situations, including those resulting from maltreatment.

NICE guideline Published November 2015

Medicines optimisation: the safe and effective use of medicines to enable the best possible outcomes (NG5)

This guideline covers safe and effective use of medicines in health and social care for people taking 1 or more medicines. It aims to ensure that medicines provide the greatest possible benefit to people by encouraging medicines reconciliation, medication review, and the use of patient decision aids.

NICE guideline Published March 2015

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

Major trauma: service delivery (NG40)

This guideline covers the organisation and provision of major trauma services in pre-hospital and hospital settings, including ambulance services, emergency departments, major trauma centres and trauma units. It aims to reduce deaths and disabilities in people with serious injuries by providing a systematic approach to the delivery of major trauma care. It does not cover services for people with burns.

NICE guideline Published February 2016

Fractures (non-complex): assessment and management (NG38)

This guideline covers assessing and managing non-complex fractures that can be treated in the emergency department or orthopaedic clinic. It aims to improve practice so that people with fractures receive the care that they need without unnecessary tests and treatments.

NICE guideline Published February 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

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

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

Child maltreatment: when to suspect maltreatment in under 18s (CG89)

This guideline covers the signs of possible child maltreatment in children and young people aged under 18 years. It aims to raise awareness and help health professionals who are not child protection specialists to identify the features of physical, sexual and emotional abuse, neglect and fabricated or induced illness.

Clinical guideline Published July 2009 Last updated October 2017

Motor neurone disease: assessment and management (NG42)

This guideline covers assessing and managing motor neurone disease (MND). It aims to improve care from the time of diagnosis, and covers information and support, organisation of care, managing symptoms and preparing for end of life care.

NICE guideline Published February 2016 Last updated July 2019

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

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

Atrial fibrillation: management (CG180)

This guideline covers diagnosing and managing atrial fibrillation in adults. It aims to ensure that people receive the best management to help prevent harmful complications, in particular stroke and bleeding.

Clinical guideline Published June 2014 Last updated August 2014

Pressure ulcers: prevention and management (CG179)

This guideline covers risk assessment, prevention and treatment in children, young people and adults at risk of, or who have, a pressure ulcer (also known as a bedsore or pressure sore). It aims to reduce the number of pressure ulcers in people admitted to secondary or tertiary care or receiving NHS care in other settings, such as primary and community care and emergency departments.

Clinical guideline Published April 2014

Type 1 diabetes in adults: diagnosis and management (NG17)

This guideline covers the care and treatment of adults (aged 18 and over) with type 1 diabetes.

NICE guideline Published August 2015 Last updated July 2016

Gallstone disease: diagnosis and management (CG188)

This guideline covers diagnosing and managing gallstone disease in adults. It aims to reduce variation in care by promoting the most effective treatments, and to improve the advice given to people with gallstone disease before and after treatment.

Clinical guideline Published October 2014

Acute heart failure: diagnosis and management (CG187)

This guideline covers diagnosing and managing acute heart failure or possible acute heart failure in people aged 18 and over. It aims to improve the immediate care of someone who is acutely unwell as a result of heart failure.

Clinical guideline Published October 2014

Dental checks: intervals between oral health reviews (CG19)

This guideline covers assigning recall intervals between oral health reviews that are appropriate to the needs of individual patients. The guideline takes account of the effect of dental checks on: people’s wellbeing, general health and preventive habits; caries incidence and avoiding restorations; periodontal health and avoiding tooth loss; and avoiding pain and anxiety. It aims to improve or maintain patients’ quality of life and reduce morbidity associated with oral and dental disease.

Clinical guideline Published October 2004

Obsessive-compulsive disorder and body dysmorphic disorder: treatment (CG31)

This guideline covers recognising, assessing, diagnosing and treating obsessive-compulsive disorder and body dysmorphic disorder in adults, young people and children (aged 8 years and older). It aims to improve the diagnosis and treatment of obsessive-compulsive disorder and body dysmorphic disorder. It includes recommendations on how families and carers may be able to support people with either of these conditions, and how they can get support for themselves.

Clinical guideline Published November 2005

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

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

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

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

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