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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

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

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

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

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-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

Velmanase alfa for treating alpha-mannosidosis [ID800]

In development [GID-HST10010] 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

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

Metreleptin for treating lipodystrophy (ID861)

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

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

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

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

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

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

Peripheral arterial disease: diagnosis and management (CG147)

This guideline covers diagnosing and managing peripheral arterial disease (PAD) in people aged 18 and over. Rapid changes in diagnostic methods, endovascular treatments and vascular services associated with new specialties in surgery and interventional radiology have resulted in considerable uncertainty and variation in practice. This guideline aims to resolve that uncertainty and variation.

Clinical guideline Published August 2012 Last updated February 2018

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

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

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

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

Spasticity in under 19s: management (CG145)

This guideline covers managing spasticity and co-existing motor disorders and their early musculoskeletal complications in children and young people (from birth up to their 19th birthday) with non-progressive brain disorders. It aims to reduce variation in practice and help healthcare professionals to select and use appropriate treatments.

Clinical guideline Published July 2012 Last updated November 2016

Osteoporosis: assessing the risk of fragility fracture (CG146)

This guideline covers assessing the risk of fragility fracture in people aged 18 and over with osteoporosis. It aims to provide guidance on the selection and use of risk assessment tools in the care of adults at risk of fragility fractures in all NHS settings.

Clinical guideline Published August 2012 Last updated February 2017

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

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

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

Mental health problems in people with learning disabilities: prevention, assessment and management (NG54)

This guideline covers preventing, assessing and managing mental health problems in people with learning disabilities in all settings (including health, social care, education, and forensic and criminal justice). It aims to improve assessment and support for mental health conditions, and help people with learning disabilities and their families and carers to be involved in their care.

NICE guideline Published September 2016

Multimorbidity: clinical assessment and management (NG56)

This guideline covers optimising care for adults with multimorbidity (multiple long-term conditions) by reducing treatment burden (polypharmacy and multiple appointments) and unplanned care. It aims to improve quality of life by promoting shared decisions based on what is important to each person in terms of treatments, health priorities, lifestyle and goals. The guideline sets out which people are most likely to benefit from an approach to care that takes account of multimorbidity, how they can be identified and what the care involves.

NICE guideline Published September 2016

Physical health of people in prison (NG57)

This guideline covers assessing, diagnosing and managing physical health problems of people in prison. It aims to improve health and wellbeing in the prison population by promoting more coordinated care and more effective approaches to prescribing, dispensing and supervising medicines.

NICE guideline Published November 2016

End of life care for infants, children and young people with life-limiting conditions: planning and management (NG61)

This guideline covers the planning and management of end of life and palliative care in for infants, children and young people (aged 0–17 years) with life-limiting conditions. It aims to involve children, young people and their families in decisions about their care, and improve the support that is available to them throughout their lives.

NICE guideline Published December 2016 Last updated July 2019

Cerebral palsy in under 25s: assessment and management (NG62)

This guideline covers diagnosing, assessing and managing cerebral palsy in children and young people from birth up to their 25th birthday. It aims to make sure they get the care and treatment they need for the developmental and clinical comorbidities associated with cerebral palsy, so that they can be as active and independent as possible.

NICE guideline Published January 2017

Sepsis: recognition, diagnosis and early management (NG51)

This guideline covers the recognition, diagnosis and early management of sepsis for all populations. The guideline committee identified that the key issues to be included were: recognition and early assessment, diagnostic and prognostic value of blood markers for sepsis, initial treatment, escalating care, identifying the source of infection, early monitoring, information and support for patients and carers, and training and education.

NICE guideline Published July 2016 Last updated September 2017

Non-Hodgkin's lymphoma: diagnosis and management (NG52)

This guideline covers diagnosing and managing non-Hodgkin's lymphoma in people aged 16 years and over. It aims to improve care for people with non-Hodgkin's lymphoma by promoting the best tests for diagnosis and staging and the most effective treatments for 6 of the subtypes. Tests and treatments covered include excision biopsy, radiotherapy, immunochemotherapy and stem cell transplantation.

NICE guideline Published July 2016