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http://igg.me/at/aku

Hi. My name is Nick Sireau, and I’m the father of two boys with a rare but devastating disease.
Alkaptonuria, or AKU for short, was the first genetic disease to be discovered more than 110 years ago. Despite this, it still has no cure, and the medical world remains largely unaware of its existence and impact on everyday life.

AKU is also known as Black Bone Disease. This is because an acid in the body accumulates at 2,000 times the normal rate, attacking the bones and turning them black and brittle. It causes severely debilitating osteoarthritis, heart disease, and other serious health complications. Patients become increasingly disabled as they get older.

But we believe we have found a cure.

And that’s why I’m reaching out to you today.

Three years ago, I gave up my job in order to devote myself, full time, to finding a cure for my kids. During that time, I’ve worked with teams in Liverpool, the United States and across Europe to plan a clinical trial.

We have found a very promising treatment: a drug called Nitisinone. If given early enough in life, it’s effectively a cure. The scientists we work with have proven this in their laboratory studies.

The real challenge is proving that this drug works in a clinical trial, in order to license nitisinone for use in AKU.

The cure will then become a reality.

All we need now is the money to bring the patients in.

But we can't do it without your help.

We’ve raised enough to pay for all the medical tests and some of the patient costs, but we’re still missing $98,000.

The rarity of AKU means patients are scattered around the world, often faced with misdiagnosis, a lack of efficient care and a lack of awareness. This means we need patients from across Europe and beyond to run a good clinical trial. Our target is to recruit 140 patients. This may seem small, but for us it's a mountain we must climb.
So please donate now.
Your money will help us to:

• Bring patients from across the world to our clinical trial centres. We believe patients shouldn't have to pay anything towards taking part and getting the help they need. 
• Allow patients to bring carers with them. There is currently no funding available for travel or accommodation for carers. As some of the patients are extremely disabled, travelling long distances by themselves can be extremely daunting.
  
• Help us care for them properly while they are on the trial, because many of them are very disabled and suffering. In some cases, we may need to hire specialist ambulances for those who are too disabled but still want to participate.
• Support the work of AKU Society, the patient group I run for people with Black Bone Disease. It will also help support our smaller sister societies across the world who are caring for our patients, by giving them the money they need to reach out to doctors and patients.
• Allow us to organise events across Europe for patients with Black Bone Disease in order to give patients everywhere access to information about the disease and the trials. For instance, in Slovakia many patients live in isolated rural communities with little access to modern communications, so its one of our only opportunities to inform them properly of the trials. 

All we need now is your support.
My vision is for our children never to have to suffer the pain of Black Bone Disease. With your help, we can make this a reality.
You can find out more about the devastating effects of Black Bone Disease by watching our AKU patient stories, updated regularly during the campaign.