On Tuesday 17th February we will be launching a new crowdfunding campaign on Indiegogo! In 2013 we raised over $100,000 thanks to your generosity and support. In this week’s blog we explain why it is time for another campaign.

In just 6 days time we will be launching a new crowdfunding campaign. As many of you will know, our last campaign was to raise money for the launch of an international clinical trial testing a promising drug called nitisinone. This trial is now well underway. We have completed recruitment, with over 135 patients taking part, and our doctors and scientists are pleased with the progress so far.

This time we need your help to raise money to research the best age for patients to begin treatment.


Why the New Campaign?

As you all know, AKU is a genetic disease, meaning it is with patients through their whole lives. From an early age urine is a dark colour, staining nappies. Unfortunately, symptoms get more serious with time. Patients develop crippling back and joint pain, resulting in many having to give up work. Older patients may need serious operations to replace or fuse their broken joints. If given at the correct age, nitisinone could prevent all the debilitating impacts of this disease.

At the moment, the clinical trial is for adults only. This is because our scientists are worried there may be side effects if the drug is given at too young an age. However, the damage caused by AKU could be starting on a microscopic level from birth.

Our research teams still don’t know exactly how the disease changes over a patient’s lifetime. Our SOFIA study will recruit patients from across Europe. Our researchers will compare patients in different age groups to assess when damage really begins. They will do this using a variety of tools, from assessing how patients walk, to taking blood, urine and ear cartilage samples. This research is very important- if we give the drug early enough, we may be able to prevent patients from ever experiencing any of the painful symptoms of AKU.

We Need Your Support

Our new campaign, ‘Help us Cure Black Bone Disease: Time is Running Out’, will launch next week, on Tuesday 17th February. To make this campaign a success we need your support! But how can you help?


Sign up to our Thunderclap now

    1. . Everyone who signs up to the Thunderclap will send out an automatic tweet or Facebook post simultaneously on the day of our campaign launch, spreading awareness over social media.

You can sign up here

    1. .

Spread the word. 

    1. When our campaign launches, spread the word to your friends, and promote it over social media.

Send us your story.

    1.  Our campaign is all about helping patients, so we want you to get involved. If you would like to write an update for us about your experience living with AKU, please send it to [email protected]
  1. Last Time

    IMG_0878 2

    Last time we were overwhelmed by your support. The original Cure Black Bone Disease campaign exceeded our funding expectations, raising in total $121,012 from 1,470 donors.

    Your money is helping patients in three key ways. Firstly we have been able to run identification campaigns across Europe, helping us find many new patients. This gave patients the opportunity to take part in the trial, and helped us recruit enough patients to make the trial a success.

    Much of the money is also being spent on patient and carer travel. Many patients are unable to travel alone, and need to have a carer with them. We wanted to ensure there was always enough money to make this possible, improving the patient experience.

    One of the most important uses of money has been for patient support. This trial is for the benefit of patients, and we want patients to feel supported every step of the way. We ensure we have continuous contact with patients, before, during, and between visits to make sure they always have someone to speak to about concerns or worries.

    Your Money


    This time your money will be used in a similar way. We want patients to be fully informed about the study, and be able to take part no matter what their circumstances, or where in Europe they live. Please help us to make this important research a reality by supporting our campaign when it launches on Tuesday 17th February.

    If you have any questions about the campaign, you can get in contact with Sorsha by emailing [email protected].

Fundraise and donate.

    1.  We are aiming to raise $30,000 (about £20,000) to support this important research. If you can help us meet this target by fundraising in your local area and donating, we would love to hear from you.