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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

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

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

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

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

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

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

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

Secondary prevention after a myocardial infarction (QS99)

This quality standard covers preventing further cardiovascular disease after a myocardial infarction (heart attack). It includes assessment and cardiac rehabilitation. It describes high-quality care in priority areas for improvement.

Quality standard Published September 2015

Atrial fibrillation (QS93)

This quality standard covers identifying and managing atrial fibrillation (including paroxysmal, persistent and permanent atrial fibrillation, and atrial flutter) in adults (aged 18 and over). It describes high-quality care in priority areas for improvement.

Quality standard Published July 2015 Last updated February 2018

Urinary tract infections in adults (QS90)

This quality standard covers diagnosing and managing urinary tract infection in adults (aged 16 and over), including adults using urinary catheters. It includes assessment, antibiotic treatment and referral for specialist assessment. It describes high-quality care in priority areas for improvement.

Quality standard Published June 2015

Pressure ulcers (QS89)

This quality standard covers preventing, assessing and managing pressure ulcers (bed sores) in adults, young people and children. It includes risk assessment and support to prevent pressure ulcers. It applies to all settings, including hospitals, care homes (with and without nursing care) and people’s own homes.

Quality standard Published June 2015

Inflammatory bowel disease (QS81)

This quality standard covers diagnosing and managing inflammatory bowel disease (Crohn’s disease and ulcerative colitis) in adults, young people and children. It describes high-quality care in priority areas for improvement.

Quality standard Published February 2015

Dyspepsia and gastro‑oesophageal reflux disease in adults (QS96)

This quality standard covers investigating and managing symptoms of dyspepsia (indigestion) and gastro-oesophageal reflux disease (heartburn or reflux) in adults (aged 18 and over). It describes high-quality care in priority areas for improvement.

Quality standard Published July 2015

Bipolar disorder in adults (QS95)

This quality standard covers recognising, assessing and managing bipolar disorder in adults (aged 18 and over) in primary and secondary care. It describes high-quality care in priority areas for improvement.

Quality standard Published July 2015

Osteoarthritis (QS87)

This quality standard covers diagnosing, assessing and managing osteoarthritis in adults. It includes treatment and support, and referral for joint surgery. It describes high-quality care in priority areas for improvement.

Quality standard Published June 2015

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

Jaundice in newborn babies under 28 days (QS57)

This quality standard covers recognising and managing jaundice in newborn babies (neonatal jaundice), from birth to 28 days, in primary care (including community care) and secondary care. It describes high-quality care in priority areas for improvement.

Quality standard Published March 2014

Sickle cell disease (QS58)

This quality standard covers managing acute painful episodes of sickle cell disease in hospital. It includes pain relief and care for children, young people and adults, from presentation in hospital until discharge. It describes high-quality care in priority areas for improvement.

Quality standard Published April 2014

Cancer services for children and young people (QS55)

This quality standard covers providing and organising cancer services for babies, children and young people (from birth to 24 years, although the upper age limit may vary). It describes high-quality care in priority areas for improvement.

Quality standard Published February 2014

Peripheral arterial disease (QS52)

This quality standard covers diagnosing and managing lower limb peripheral arterial disease in adults (aged 18 and over). It describes high-quality care in priority areas for improvement.

Quality standard Published January 2014

Autism (QS51)

This quality standard covers health and social care services for adults, young people and children with autism. It includes assessment and diagnosis of autism spectrum disorders, and care and support for people diagnosed with an autism spectrum disorder. It describes high-quality care in priority areas for improvement.

Quality standard Published January 2014

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

Mental wellbeing of older people in care homes (QS50)

This quality standard covers the mental wellbeing of older people (aged 65 and over) receiving care in care homes (including residential and nursing accommodation, day care and respite care). It focuses on support for people to improve their mental wellbeing so that they can stay as well and independent as possible. It describes high-quality care in priority areas for improvement.

Quality standard Published December 2013

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

Acute kidney injury (QS76)

This quality standard covers preventing, detecting and managing acute kidney injury in adults, young people and children. Acute kidney injury is sudden damage to the kidneys, which can sometimes result in kidney failure. The quality standard includes assessment by non-specialists and when to refer to specialist services. It describes high-quality care in priority areas for improvement.

Quality standard Published December 2014

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

Idiopathic pulmonary fibrosis in adults (QS79)

This quality standard covers managing idiopathic pulmonary fibrosis (gradual scarring of the lungs) in adults. It includes diagnosis by a multidisciplinary team, managing symptoms and palliative care. It describes high-quality care in priority areas for improvement.

Quality standard Published January 2015

Sarcoma (QS78)

This quality standard covers providing and organising services for people with sarcoma. It includes pathways for referral and diagnosis, treatment and support for adults, young people and children with sarcoma. It describes high-quality care in priority areas for improvement.

Quality standard Published January 2015