In the UK around 850,000 people suffer from dementia. Most of us will know of someone who suffers or is affected by this disease. It mainly affects people over the age of 65, which amounts to around one person in every 14 in the age group. The word ‘dementia’ describes a set of symptoms that may include memory loss and difficulties with thinking, problem solving or language. Changes are often small to start with, but for someone with dementia they have become severe enough to affect daily life. A person with dementia may also experience changes in their mood or behaviour. Dementia is caused when diseases, such as Alzheimer’s disease or a series of strokes damage the brain. Alzheimer’s disease is the most common cause of dementia, but not the only one. The specific symptoms that someone with dementia experiences will depend on the parts of the brain that are damaged and the disease that is causing the dementia.
Dementia also affects younger people. There are more than 42,000 people in the UK under 65 with dementia. ‘Harvey and the Brave Little Soldiers’ – early onset Alzheimer’s Niemann-Pick diseases – are a group of rare inherited storage disorders that can affect both children and adults. Brave little soldiers raise vital funds for research and support in helping families affected. NP-C is an extremely scary and uncertain disease that unfairly affects our most vulnerable children.
Harvey was diagnosed with the rare neurogenetic disease Niemann-Pick Type C (NP-C). This is a very rare progressive disorder characterised by an inability of the body to transport cholesterol and other fatty substances (lipids) inside of the cells. This leads to an abnormal accumulation of these substances within various tissues of the body, including brain tissue. The accumulation of these substances damages the affected areas. Individuals with NP-C can have an onset of symptoms at different ages and they can vary from person to person.
There is currently no cure for Alzheimer’s or Niemann-Pick Disease Type C (NP-C). The incidence of NP-C is widely reported at 1 in 120,000, although recent evidence suggests this may be an under-estimate. A child with NP-C will often experience quick and progressive mental and physical deterioration over the course of several years – similar to Alzheimer’s disease hence its nickname “Childhood Alzheimer’s”.
Like Alzheimer’s, NP-C currently has no cure and leads to death. What makes this particular disease so difficult to diagnose is the symptoms are non-specific to the disease and will vary from person to person. The age of onset and rate of disease progression can vary greatly from person to person; for example some children develop neurological symptoms early in childhood, whereas others may remain symptom free for a number of years. Patients benefit from palliative treatments (individual medications that will help to treat the symptoms related to the condition). Occupational therapy can be used to help with posture, speech and movement.
In recent years, thanks to the hard work of individuals and charities such as the Alzheimers Society and Harvey and the Brave Little Soldiers more research than ever is happening right now! There are a number of clinical trials currently taking place investigating new therapeutic options for patients with NP-C. Medical trials cannot take place without vital funding. NPUK do NOT receive any funding from the government and rely solely on kind donations from people such as yourselves.
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