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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

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

Safe staffing for nursing in adult inpatient wards in acute hospitals (SG1)

This guideline covers organisational and managerial approaches to safe nurse staffing of inpatient wards for people aged 18 and over in acute hospitals. It aims to ensure that patients receive the nursing care they need, regardless of the ward to which they are allocated, the time of the day, or the day of the week.

Safe staffing guideline Published July 2014

Developing and updating local formularies (MPG1)

This guideline covers good practice for developing and updating local formularies in line with statutory requirements. It supports developing formularies that reflect local needs, reduce variation in prescribing, and allow rapid adoption of new medicines and treatments.

Medicines practice guideline Published March 2014 Last updated October 2015

Patient group directions (MPG2)

This guideline covers good practice for developing, authorising, using and updating patient group directions. It also offers advice on deciding whether a patient group direction is needed.

Medicines practice guideline Published August 2013 Last updated March 2017

Managing medicines in care homes (SC1)

This guideline covers good practice for managing medicines in care homes. It aims to promote the safe and effective use of medicines in care homes by advising on processes for prescribing, handling and administering medicines. It also recommends how care and services relating to medicines should be provided to people living in care homes.

Social care guideline Published 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

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