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Spinal muscular atrophy (SMA) is a neuromuscular disorder that results in progressive muscle weakness with onset in childhood and adolescence. There are three main types of SMA. Drug treatment for SMA type I is discussed in a separate Cochrane review. This review is of drug treatment for SMA types II and III. Both of these reviews were first published in 2009 and are now updated. The age of onset of SMA type II is between six and 18 months. Children with SMA type II will never be able to walk without support; they survive beyond two years and may live into adolescence or longer. The age of onset of SMA III, also known as Kugelberg‐Welander disease, is after 18 months. Children with SMA type III develop the ability to walk at some time and their life expectancy is normal. From six randomised controlled trials, there is no evidence for a significant effect on the disease course when patients with SMA types II and III are treated with creatine (55 participants), phenylbutyrate (107 participants), gabapentin (84 participants), thyrotropin releasing hormone (9 participants), hydroxyurea (57 participants) or combination therapy with valproate and acetyl‐L‐carnitine (61 participants). The risk of bias of the included trials was systematically analysed and none of the studies were completely free of bias. Thus, there is still no known efficacious drug treatment for SMA types II and III.

Cochrane Database of Systematic Reviews: Plain Language Summaries [Internet] - John Wiley & Sons, Ltd.

Version: April 18, 2012

Spinal muscular atrophy (SMA) is a severe neuromuscular disease with onset in childhood and adolescence that results in progressive muscle weakness. There are three main types of SMA. Drug treatment for SMA types II and III is discussed in a separate Cochrane review. The age of onset of SMA type I, also known as Werdnig‐Hoffmann disease, is before six months. Children with SMA type I will never be able to sit without support and usually die by the age of two years. It is one of the most important causes of death due to a genetic disease in childhood. There was only one small randomised trial in the original review, which assessed the efficacy of riluzole for 10 children with SMA type I. In this trial all three children in the placebo group died, but three of the seven children treated with riluzole were still alive at the ages of 30, 48 and 64 months. However, none of the children in the riluzole or placebo group developed the ability to roll, sit or stand. For several reasons the overall quality of the study was low, mainly because the study was too small to detect an effect and there were baseline differences that resulted in risk of bias. Evidence is insufficient to recommend riluzole for SMA type I. No further trials were identified for this 2011 update. No drug treatment has been shown to have significant efficacy for SMA type I.

Cochrane Database of Systematic Reviews: Plain Language Summaries [Internet] - John Wiley & Sons, Ltd.

Version: April 18, 2012

Motor neurone disease (MND) is a neurodegenerative condition that affects the brain and spinal cord. MND is characterised by the degeneration of primarily motor neurones, leading to muscle weakness.

NICE Guideline - National Clinical Guideline Centre (UK).

Version: February 2016

In summary, a large number of women in the UK experience menopausal symptoms which, in many cases, can significantly affect their quality of life. It is probable that a minority of these women seek medical treatment and for those who do there is considerable variation in the help available, with many being told that the symptoms will get better with time. Since symptoms may often continue for 7 years or more, this advice is inappropriate and help should be offered where possible. Women need to know about the available options and their risks and benefits, and be empowered to become part of the decision-making process.

NICE Guideline - National Collaborating Centre for Women's and Children's Health (UK).

Version: November 12, 2015

Heavy menstrual bleeding (HMB) has an adverse effect on the quality of life of many women. It is not a problem associated with significant mortality. Many women seek help from their general practitioners and it is a common reason for referral into secondary care.

NICE Clinical Guidelines - National Collaborating Centre for Women's and Children's Health (UK).

Version: January 2007

The study found limited evidence on the effectiveness of the various types of orthoses used within the NHS to manage patients with neuromuscular and central nervous system conditions, especially in relation to the outcomes that are important to patients.

Health Technology Assessment - NIHR Journals Library.

Version: July 2016

This guideline covers the physical, emotional, social and spiritual elements of end of life care, and focuses on improving the child or young person’s quality of life and supporting their family and carers. There are, for instance, recommendations on managing distressing symptoms and providing care and bereavement support after death. Recommendations have also been made about how services should be delivered. The guideline is aimed at all providers of paediatric end of life care, whatever their level of practise, and also for children and young people with life-limiting conditions and their parents or carers.

NICE Guideline - National Guideline Alliance (UK).

Version: December 2016

The study found that obesity [as measured using body mass index (BMI)] was found to persist from adolescence to adulthood and is associated with an increased risk of adult disease. However, increased BMI is not good at identifying which children (obese or healthy weight) will have obesity-related diseases in adulthood.

Health Technology Assessment - NIHR Journals Library.

Version: June 2015

Clinical guidelines have been defined as ‘systematically developed statements which assist clinicians and patients in making decisions about appropriate treatment for specific conditions’. This guideline has been developed with the aim of providing guidance on the care of children with bronchiolitis.

NICE Guideline - National Collaborating Centre for Women's and Children's Health (UK).

Version: June 2015

The study found that there are a number of impacts and likely impacts of the Cochrane Reviews on research targeting and health-care policy, with less evidence of a direct impact on clinical practice and the organisation and delivery of NHS services.

Health Technology Assessment - NIHR Journals Library.

Version: April 2015

The study found agreement between young people, parents and professional regarding the most important health outcomes regarding neurodisability for identification through patient-reported outcome measures (PROMs). A systematic review and focus groups were used to examine the effectiveness of PROMs for measuring the specified outcomes, but found that none of the candidate PROMs in the review adequately captured all of these; in addition, there is inadequate evidence that candidate PROMs are psychometrically robust for use with children with neurodisability. Further research is needed to test potential PROMs for use across this population.

Health Services and Delivery Research - NIHR Journals Library.

Version: May 2014

This study found that no definitive conclusions can be made regarding either the clinical effectiveness or the cost-effectiveness of the use of SeptiFast, SepsiTest or IRIDICA for the identification of bloodstream bacteria and fungi. Clinical judgement suggests that the tests are likely to be beneficial to patients but this needs to be proven in appropriate studies.

Health Technology Assessment - NIHR Journals Library.

Version: June 2016

The guideline covers adults and children (from birth) with lower urinary tract dysfunction resulting from neurological disease or injury.

NICE Clinical Guidelines - National Clinical Guideline Centre (UK).

Version: August 2012

It is almost 200 years since James Parkinson described the major symptoms of the disease that came to bear his name. Slowly but surely our understanding of the disease has improved and effective treatment has been developed, but Parkinson’s disease remains a huge challenge to those who suffer from it and to those involved in its management. In addition to the difficulties common to other disabling neurological conditions, the management of Parkinson’s disease must take into account the fact that the mainstay of pharmacological treatment, levodopa, can eventually produce dyskinesia and motor fluctuation. Furthermore, there are a number of agents besides levodopa that can help parkinsonian symptoms, and there is the enticing but unconfirmed prospect that other treatments might protect against worsening neurological disability. Thus, a considerable degree of judgement is required in tailoring individual therapy and in timing treatment initiation. It is hoped that this guideline on Parkinson’s disease will be of considerable help to those involved at all levels in these difficult management decisions. The guideline has been produced using standard NICE methodology and is therefore based on a thorough search for best evidence.

NICE Clinical Guidelines - National Collaborating Centre for Chronic Conditions (UK).

Version: 2006

For many people faecal incontinence is the result of a complex interplay of contributing factors, many of which can co-exist. Some may be relatively simple to reverse.

NICE Clinical Guidelines - National Collaborating Centre for Acute Care (UK).

Version: 2007

This guidance is a partial update of National Institute for Health and Care Excellence (NICE) clinical guideline 40 (published October 2006) and will replace it.

NICE Clinical Guidelines - National Collaborating Centre for Women's and Children's Health (UK).

Version: September 2013

Motor neurone disease (MND) is a fatal neurodegenerative disease. It is characterised by the onset of symptoms and signs of degeneration of primarily the upper and lower motor neurones. This leads to progressive weakness of the bulbar, limb, thoracic and abdominal muscles. Respiratory muscle weakness resulting in respiratory impairment is a major feature of MND, and is a strong predictor of quality of life and survival. Non-invasive ventilation can improve symptoms and signs related to respiratory impairment and hence survival.

NICE Clinical Guidelines - National Institute for Health and Clinical Excellence (UK).

Version: July 2010

Multiple sclerosis (MS) is an acquired chronic immune-mediated inflammatory condition of the central nervous system (CNS), affecting both the brain and spinal cord. It affects approximately 100,000 people in the UK. It is the commonest cause of serious physical disability in adults of working age.

NICE Clinical Guidelines - National Clinical Guideline Centre (UK).

Version: October 2014

Osteoarthritis refers to a clinical syndrome of joint pain accompanied by varying degrees of functional limitation and reduced quality of life. It is the most common form of arthritis, and one of the leading causes of pain and disability worldwide. The most commonly affected peripheral joints are the knees, hips and small hand joints. Although pain, reduced function and effects on a person’s ability to carry out their day-to-day activities can be important consequences of osteoarthritis, pain in itself is of course a complex biopsychosocial issue, related in part to person expectations and self-efficacy, and associated with changes in mood, sleep and coping abilities. There is often a poor link between changes on an X-ray and symptoms: minimal changes can be associated with a lot of pain and modest structural changes to joints oftencan occur without with minimal accompanying symptoms. Contrary to popular belief, osteoarthritis is not caused by ageing and does not necessarily deteriorate. There are a number of management and treatment options (both pharmacological and non-pharmacological), which this guideline addresses and which offer effective interventions for control of symptoms and improving function.

NICE Clinical Guidelines - National Clinical Guideline Centre (UK).

Version: February 2014

Cancer is an important condition, both in terms of the number of people affected and the impacts on those people and the people close to them. Around one third of a million new cancers are diagnosed annually in the UK, across over 200 different cancer types. Each of these cancer types has different presenting features, though there may be overlap. More than one third of the population will develop a cancer in their lifetime. Although there have been large advances in treatment and survival, with a half of cancer sufferers now living at least ten years after diagnosis, it remains the case that more than a quarter of all people alive now will die of cancer.

NICE Guideline - National Collaborating Centre for Cancer (UK).

Version: June 2015

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