Thursday, October 31, 2013

Cambridge, UK, 31 October 2013 – A patient group has used the crowdfunding website Indiegogo to raise $120,000 to launch an international clinical trial to treat Black Bone Disease, the first genetic disease ever discovered.

The campaign, called Help Us Cure Black Bone Disease, ran from 1 September to 21 October 2013 and set an initial target of raising $98,000 in six weeks. In the first 11 days post-launch, it had already raised $25,570 – over 25% of its goal. By its end, the figure was higher than $120,000, exceeding the campaign’s goal by 20%.

Dr Nicolas Sireau, Chairman of the AKU Society, the patient group that ran the campaign, said: “This funding will enable us to recruit patients across Europe into clinical trials of nitisinone. As the world’s first potential treatment for AKU, nitisinone could transform patients’ lives.”

The campaign’s Indiegogo page attracted 28,598 visits over 50 days, leading to 1,470 donors from 40 countries donating a total of $98,685. Including offline donations, the grand total raised was $121,002.

The campaign was featured in major national UK media, such as the Sunday Express, BBC Radio 5, the MailOnline, the Telegraph and the Daily Mirror.

Beyond the UK, this achievement has resonated with organisations such as EURORDIS, the European Rare Disease Organisation.

Denis Costello, EURORDIS’s Senior Web Communications Manager, said: “This will inspire rare disease patient groups globally. Crowdfunding offers a unique hope to many small networks of patients and families to kick-start or advance research into diseases which have up until now found it difficult to progress beyond even the most basic research. In the case of AKU, this community is one step closer to long-term access to a life-changing treatment.”

The clinical trials, called DevelopAKUre, are led by the Royal Liverpool University Hospital, University of Liverpool and 11 other partners. The AKU Society leads on patient recruitment and support. While sufficient money had been raised for the medical research, the AKU Society needed donations to cover patient travel and patient support throughout the five years of the programme.

Read about the AKU Society’s Indiegogo campaign at:


About alkaptonuria (AKU) / black bone disease
Alkaptonuria (AKU) was the first genetic disease ever discovered. It causes a severe form of osteoarthritis, cardiovascular problems, kidney stones and prostate stones. Symptoms typically start when people are in their 30s. Without treatment, patients develop serious joint disease. Traditional treatments include pain management and joint replacement surgeries, but these do little to slow progression of disease. Most AKU patients will need joint replacements at some points in their life, with many having over 10 such procedures.
For more information please visit:

About the AKU Society
The AKU Society is a patient group dedicated to helping patients diagnosed with alkaptonuria (AKU). It helps and supports patients, raises awareness of AKU and supports research into its treatment. The AKU Society is based in Cambridge, UK.
For more information please visit:

About the DevelopAKUre clinical trials DevelopAKUre is a series of clinical trials designed to assess whether a drug called nitisinone is an effective treatment for AKU. It is led by a consortium of 13 partners across Europe, including the coordinator (Royal Liverpool University Hospital), the sponsor (University of Liverpool) and the AKU Society. It is taking place at the National AKU Centre in Liverpool, UK; Hopital Necker in Paris, France; and the National Institute of Rheumatic Disease in Piestany, Slovakia. The trials are principally funded by a €6 million grant from the European Commission (EC), with further funding from the Indiegogo crowdfunding campaign covering patient and carer expenses, and additional support work. For more information please visit:
EURORDIS is a non-governmental patient-driven alliance representing 593 rare disease patient organisations in 54 countries. It is the voice of 30 million people affected by rare diseases throughout Europe. Further details concerning EURORDIS and rare diseases are available at:

About Indiegogo and crowdfunding
Crowdfunding is a new method of donating to good causes. It relies on small donations from a large number of people in order to reach specific funding goals. Crowdfunding has become a popular way of donating online, and several websites exist to help individuals, charities and companies make their projects known to the public.

Indiegogo is one of the world’s leading crowdfunding platforms, having raised millions of dollars for thousands of campaigns worldwide. Crowdfunding success stories on Indiegogo include the Scanadu Scout, a scanner attachment for a smartphone that helps to monitor its user’s health. Earlier this year, the Scanadu campaign exceeded its $100,000 goal to raise over $1.6 million.

Oliver Timmis, Head of Projects
Email: ol****@ak********.org
Mobile: +44 (0)7799 037726
Office: +44 (0)1223 322897

AKU Society
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