Velmanase alfa for treating alpha-mannosidosis [ID800]: evaluation consultation
We are listening to your views on this Highly specialised technology. Comments close 16 August 2022.
We are listening to your views on this Highly specialised technology. Comments close 16 August 2022.
Evidence-based recommendations on atidarsagene autotemcel (Libmeldy) for treating metachromatic leukodystrophy in children
Evidence-based recommendations on inotersen (Tegsedi) for stage 1 and stage 2 polyneuropathy in adults with hereditary transthyretin amyloidosis
Evidence-based recommendations on burosumab (Crysvita) for treating X-linked hypophosphataemia (XLH) in children and young people
Evidence-based recommendation on Strimvelis for adenosine deaminase deficiency–severe combined immunodeficiency
Evidence-based recommendations on asfotase alfa (Strensiq) for treating paediatric-onset hypophosphatasia in adults and children
Evidence-based recommendations on eliglustat (Cerdelga) for treating type 1 Gaucher disease in adults
Evidence-based recommendations on cerliponase alfa (Brineura) for treating neuronal ceroid lipofuscinosis type 2 in children
Evidence-based recommendations on voretigene neparvovec (Luxturna) for RPE65-mediated inherited retinal dystrophies in people with vision loss caused by inherit
Evidence-based recommendations on patisiran (Onpattro) for treating hereditary transthyretin amyloidosis in adults with stage 1 and stage 2 polyneuropathy
Evidence-based recommendations on givosiran (Givlaari) for treating acute hepatic porphyria in adults and young people aged 12 and over
Evidence-based recommendations on migalastat (Galafold) for treating Fabry disease in people over 16
Evidence-based recommendations on ataluren (Translarna) for treating Duchenne muscular dystrophy with a nonsense mutation in the dystrophin gene in people aged
Evidence-based recommendations on volanesorsen (Waylivra) for treating familial chylomicronaemia syndrome in adults
Evidence-based recommendations on metreleptin (Myalepta) for treating lipodystrophy in children and adults
Evidence-based recommendations on eculizumab (Soliris) for treating atypical haemolytic uraemic syndrome in adults and children
Evidence-based recommendations on onasemnogene abeparvovec (Zolgensma) for treating spinal muscular atrophy in babies
Evidence-based recommendations on elosulfase alfa (Vimizim) for treating mucopolysaccharidosis type 4A in people of all ages
Evidence-based recommendations on selumetinib (Koselugo) for treating symptomatic and inoperable plexiform neurofibromas associated with type 1 neurofibromatosis in children aged 3 and over
Evidence-based recommendations on setmelanotide (Imcivree) for treating obesity caused by LEPR or POMC deficiency in people 6 years and over
Evidence-based recommendations on odevixibat (Bylvay) for treating progressive familial intrahepatic cholestasis in people 6Â months and older
This guideline covers routine preoperative tests for people aged over 16 who are having elective surgery. It aims to reduce unnecessary testing by advising which tests to offer people before minor, intermediate and major or complex surgery, taking into account specific comorbidities (cardiovascular, renal and respiratory conditions and diabetes and obesity). It does not cover pregnant women or people having cardiothoracic procedures or neurosurgery.
This guideline covers diagnosing, managing and referring infants and young children younger than 5 years who present with acute diarrhoea (lasting up to 14 days) with or without vomiting. It aims to improve the diagnosis and management of infective gastroenteritis and appropriate escalation of care.
This guideline covers the assessment and early management of head injury in children, young people and adults. It promotes effective clinical assessment so that people receive the right care for the severity of their head injury, including referral directly to specialist care if needed.
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.
This guideline covers care for people aged 18 and over with common mental health problems, with a focus on primary care. It aims to improve access to services for adults and how mental health problems are identified and assessed, and makes recommendations on local care pathways.
This guideline covers assessing and managing osteoarthritis in adults. It covers both pharmacological and non-pharmacological treatments. It promotes effective treatment options to control joint pain and improve function in people with osteoarthritis.
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.
This guideline covers diagnosing and managing drug allergy in all age groups. It aims to make it easier for professionals to tell when someone is having an allergic reaction, by specifying the key signs and patterns to look out for. It also makes recommendations on improving people’s understanding of their drug allergies, and ensuring these are recorded properly in their medical records.
This guideline covers identifying, assessing and managing obesity in children (aged 2 years and over), young people and adults. It aims to improve the use of bariatric surgery and very-low-calorie diets to help people who are obese to reduce their weight.
This guideline covers recognising and managing psychosis and schizophrenia in adults. It aims to improve care through early recognition and treatment, and by focusing on long-term recovery. It also recommends checking for coexisting health problems and providing support for family members and carers.
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.
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.
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.
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.
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.
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.
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.
This guideline covers the general principles for managing intravenous (IV) fluid therapy in hospital inpatients aged 16 and over with a range of conditions. It aims to help prescribers understand the optimal amount and composition of IV fluids to be administered and the best rate at which to give them, to improve fluid prescribing and outcomes among people in hospital. It does not cover pregnant women, and those with severe liver or renal disease, diabetes or burns.
This guideline covers the care of healthy women and their babies, during labour and immediately after the birth. It focuses on women who give birth between 37 and 42 weeks of pregnancy (‘term’). The guideline helps women to make an informed choice about where to have their baby. It also aims to reduce variation in areas of care such as fetal monitoring during labour and management of the third stage of labour.
This guideline covers assessment, diagnosis and referral for people over 16 who have had a transient loss of consciousness (TLoC; also called a blackout). It aims to improve care for people with TLoC by specifying the most effective assessments and recommending when to refer to a specialist.
This guideline covers diagnosing and managing Lyme disease. It aims to raise awareness of when Lyme disease should be suspected and ensure that people have prompt and consistent diagnosis and treatment. It does not cover preventing Lyme disease.
This guideline covers organising and delivering emergency and acute medical care for people aged over 16 in the community and in hospital. It aims to reduce the need for hospital admissions by giving advanced training to paramedics and providing community alternatives to hospital care. It also promotes good-quality care in hospital and joint working between health and social services.
This guideline covers diagnosing, monitoring and managing any type of primary brain tumour or brain metastases in people aged 16 or over. It aims to improve diagnosis and care, including standardising the care people have, how information and support are provided, and palliative care.
This guideline covers assessing and managing oesophago-gastric cancer in adults, including radical and palliative treatment and nutritional support. It aims to reduce variation in practice through better organisation of care and support, and improve quality of life and survival by giving advice on the most suitable treatments depending on cancer type, stage and location.
This guideline covers diagnosing, monitoring and managing asthma in adults, young people and children. It aims to improve the accuracy of diagnosis, help people to control their asthma and reduce the risk of asthma attacks. It does not cover managing severe asthma or acute asthma attacks.
This guideline covers surgical treatment of children younger than 12 years who have fluid buildup behind their eardrum (otitis media, also known as ‘glue ear’). It aims to improve hearing and quality of life in children with otitis media.
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.
This guideline covers preventing infective endocarditis (IE) in children, young people and adults. It focuses on people at increased risk of infective endocarditis undergoing dental, gastrointestinal tract, genitourinary and respiratory tract procedures.
This guideline covers assessing and diagnosing recent chest pain in people aged 18 and over and managing symptoms while a diagnosis is being made. It aims to improve outcomes by providing advice on tests (ECG, high-sensitivity troponin tests, multislice CT angiography, functional testing) that support healthcare professionals to make a speedy and accurate diagnosis.