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ALD Life operates to raise awareness of rare diseases and specically ALD Adrenoleukodystrophy (which affects children) and

d AMN Adrenomyeloneuropathy (which affects adults). For these diseases at the moment there is no cure ! Please, support ALD Life to raise awareness about the condition of patients and families with these terrible diseases. If you want to donate, you can

Rare Diseases?
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How many people are affected by a rare disease in the UK? Less than 500,000 About 1 million Above 3.5 million For the majority of patients, how long does it take to receive a diagnosis of a rare disease? Less than 1 year 1 to 5 years 5 years over 20 years Being a boy with A-symptomatic ALD means... That the boy has no symptoms and so he is well That the boy shows no symptoms but he has to be monitored for a certain period of time That the boy shows no symptoms but he has to be monitored constantly for the rest of his life How long does it take for a healthy boy affected by ALD to become fully dependent? Few months At least 1 year From 1 to 5 years

Text RARE29 10 to 70070


Send a cheque to ALD Life, PO BOX 43642, London SE22 ORG A huge thank you for your time, wanting to learn more about rare disease, and thanks for your support of ALD Life from all our patients and our families! ALD Life www.aldlife.org

rs Quiz Answe

boy will have a bone marrow transplant, the only treatment known to stop the onset of symptomatic ALD. However this only works if the symptoms are caught early through regular MRI scans.

Above 3.5 million 1 in 17 people will be affected by a rare disease at some point in their life. This amounts to approximately 3.5 million people in the UK. There are over 6,000 recognised rare diseases. Collectively rare diseases are not rare.

All three! There is no set time pattern for the development of symptoms, but most commonly boys lose their abilities within months of diagnosis. This is what happened to Alex. He was a normal, bright, healthy, outgoing child until the age of seven. His rst symptoms where naughty behaviour and inattentiveness in class. These were shortly followed by poor hand to eye co-ordination and episodes of confusion. He became fully dependent in six months. Alex is the son of Sara Hunt, who decided to form ALD Life in 2003. Saras objectives are: to support other families and patients in similar circumstances with practical advice, information and emotional support to raise as much money as possible as all the research into the conditions is charity and private funded

1 to 5 years Almost 30% of patients received a diagnosis of a genetic rare disease after 1 to 5 years of test and exams. 19% of patients waited more than 5 years and up to 20 years. Patients and families affected by rare diseases wait far too long for a correct diagnosis and a worrying number receive incorrect diagnoses. This is due to the low level of awareness of rare diseases among healthcare professionals. Patient support groups play a fundamental role in helping patients to receive a correct diagnosis.

That the boy shows no symptoms but has to be monitored consistently for the rest of his life Boys with asymptomatic ALD carry the potential to develop the devastating effects of symptomatic ALD. To prevent this, when it is known that a boy has the ALD gene he is monitored closely to detect the rst signs that symptomatic ALD is developing. As soon as this is established the