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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

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

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

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

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

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

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

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

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

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

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

Balloon dilation for eustachian tube dysfunction

In development [GID-IPG10125] Expected publication date: 11 December 2019

Interventional procedures guidance In development

Balloon cryoablation for Barrett's oesophagus

In development [GID-IPG10127] Expected publication date: 11 December 2019

Interventional procedures guidance In development

Balloon cryoablation for squamous dysplasia of the oesophagus

In development [GID-IPG10139] Expected publication date: 11 December 2019

Interventional procedures guidance In development

Hypertension in pregnancy (QS35)

This quality standard covers diagnosing and managing hypertension (high blood pressure) and pre-eclampsia during pregnancy, labour and birth. It also covers advice for women with hypertension who may become pregnant and postnatal care for women who have had hypertension or pre-eclampsia. It describes high-quality care in priority areas for improvement.

Quality standard Published July 2013 Last updated July 2019

Epilepsy in children and young people (QS27)

This quality standard covers diagnosing, treating and managing epilepsy and seizures in children and young people (under 18). It describes high-quality care in priority areas for improvement.

Quality standard Published February 2013

Epilepsy in adults (QS26)

This quality standard covers diagnosing, treating and managing epilepsy and seizures in adults (aged 18 and older). It describes high-quality care in priority areas for improvement.

Quality standard Published February 2013

Nutrition support in adults (QS24)

This quality standard covers care for adults (aged 18 and over) who are malnourished or at risk of malnutrition in hospital or in the community. It includes identifying people at risk of malnutrition and providing nutrition support, including dietary changes and artificial nutrition support given through feeding tubes (enteral nutrition) or directly into a vein (parenteral nutrition). It describes high-quality care in priority areas for improvement.

Quality standard Published November 2012

Antenatal care (QS22)

This quality standard covers care for healthy women and their babies during pregnancy (up to 42 weeks). It covers routine antenatal care in primary, community and hospital settings. It describes high-quality care in priority areas for improvement.

Quality standard Published September 2012 Last updated April 2016

Neonatal specialist care (QS4)

This quality standard covers the care of babies who need specialist neonatal services, including transfer services. Specialist neonatal services deliver special, high-dependency, intensive or surgical care to newborn babies. It describes high-quality care in priority areas for improvement.

Quality standard Published October 2010

Self‑harm (QS34)

This quality standard covers the initial management of self-harm and the provision of longer-term support for children and young people (aged 8 to 18) and adults (aged 18 and over) who self-harm. It describes high-quality care in priority areas for improvement.

Quality standard Published June 2013

Looked-after children and young people (QS31)

This quality standard covers the health and wellbeing of looked-after children and young people (from birth to 18 years) and care leavers (including young people planning to leave care or under leaving care provisions). It is for all settings and services that work with and care for looked-after children and young people, wherever they are living (for example, with family or friends, with foster families or in residential care). It describes high-quality care in priority areas for improvement.

Quality standard Published April 2013

Patient experience in adult NHS services (QS15)

This quality standard covers improving the quality of the patient experience for people who use adult NHS services. It describes high-quality care in priority areas for improvement.

Quality standard Published February 2012 Last updated July 2019

Stroke in adults (QS2)

This quality standard covers diagnosing and managing stroke in adults (over 16). It includes diagnosis, initial management, acute-phase care, rehabilitation and long-term support for people with stroke. It describes high-quality care in priority areas for improvement.

Quality standard Published June 2010 Last updated April 2016

Ovarian cancer (QS18)

This quality standard covers the recognition and initial management of ovarian cancer. It describes high-quality care in priority areas for improvement.

Quality standard Published May 2012

Surgical site infection (QS49)

This quality standard covers preventing and treating surgical site infections. It covers adults, young people and children having a surgical procedure that involves a cut to the skin in all healthcare settings. It describes high-quality care in priority areas for improvement.

Quality standard Published October 2013

Heavy menstrual bleeding (QS47)

This quality standard covers assessing and managing heavy menstrual bleeding (also called menorrhagia or heavy periods). It includes assessment, diagnosis, and drug and surgical treatment options, including treatments for uterine fibroids associated with heavy menstrual bleeding. It describes high-quality care in priority areas for improvement.

Quality standard Published September 2013 Last updated March 2018

Atopic eczema in under 12s (QS44)

This quality standard covers diagnosing and managing atopic eczema in children under 12. It describes high-quality care in priority areas for improvement.

Quality standard Published September 2013

Headaches in over 12s (QS42)

This quality standard covers diagnosing and managing common types of headache in adults and young people (aged 12 and over). It includes tension-type headache, migraine, cluster headache and medication overuse headache. It describes high-quality care in priority areas for improvement.

Quality standard Published August 2013

Gallstone disease (QS104)

This quality standard covers diagnosing and managing gallstones in adults. It also includes managing complications of gallstones, such as an inflamed or infected gallbladder (cholecystitis), blocked and infected bile ducts (cholangitis), and an inflamed pancreas (pancreatitis). It describes high-quality care in priority areas for improvement.

Quality standard Published December 2015

Cardiovascular risk assessment and lipid modification (QS100)

This quality standard covers identifying and assessing cardiovascular risk in adults (aged 18 and over) and treatment to prevent cardiovascular disease. It describes high-quality care in priority areas for improvement.

Quality standard Published September 2015

Chronic obstructive pulmonary disease in adults (QS10)

This quality standard covers assessing, diagnosing and managing chronic obstructive pulmonary disease (COPD). It describes high-quality care in priority areas for improvement.

Quality standard Published July 2011 Last updated February 2016

Acute coronary syndromes in adults (QS68)

This quality standard covers diagnosing and managing acute coronary syndromes in adults (aged 18 and over). Acute coronary syndromes are medical emergencies that include myocardial infarction (heart attack) and unstable angina (unexpected, severe chest pain). It describes high-quality care in priority areas for improvement.

Quality standard Published September 2014

Constipation in children and young people (QS62)

This quality standard covers the diagnosis and management of constipation of unknown cause in babies, children and young people (from birth to 18 years). It describes high-quality care in priority areas for improvement.

Quality standard Published May 2014

Inducing labour (QS60)

This quality standard covers the induction of labour in hospital outpatient or inpatient settings. It includes advice and care for pregnant women who are considering or having induction of labour. It describes high-quality care in priority areas for improvement.

Quality standard Published April 2014

Diabetes in adults (QS6)

This quality standard covers care and treatment for adults with diabetes. It includes preventing type 2 diabetes, managing type 1 and type 2 diabetes, diabetes-related foot care and diabetes education programmes. It describes high-quality care in priority areas for improvement.

Quality standard Published March 2011 Last updated August 2016