I’ve just been reading through the consultation document for the UK plan on rare diseases. Despite being well written, it’s rather disappointing.
The UK hasn’t yet had a plan or a strategy for rare diseases, unlike countries such as France, which is now on its second national plan for rare diseases. So it was with trepidation [...]
I came home late from London last night. We’d just had a brainstorm with a group of friends to think about how to build a strong rare disease movement in the UK. As you’ll see from my previous posts, I’m convinced that more can be done to create a mass social movement for rare diseases [...]
Yesterday, I was not happy. I’d been talking to a major funder, trying to find out more about whether we could apply for funding for our fast moving scientific research and upcoming clinical trials for Alkaptonuria (AKU), the rare genetic disease affecting my two sons. The funder was very helpful in explaining the different funding [...]
What are the key factors that could explain the rise of the rare disease movement and help us understand how to make it grow faster and more effectively?
A few years ago I finished my PhD in social movement studies, a sub-group of sociology that studies why social movements rise and fall. My PhD was on [...]
We’ve just finished our fourth international conference on AKU (Alkaptonuria), here in Cambridge. It was a huge success, although it raised, once again, the questions we face as we work to find treatments for rare diseases.
Sixty scientists, industry representatives, patients and relatives attended, including from the US, Slovakia, Jordan, France, Italy, the UK, the Netherlands, [...]
I’m just back from the Partnering for Cures conference in New York (www.partneringforcures.org), where we were presenting our international findAKUre project on Alkaptonuria and trying to raise funds for our plans for new clinical trials for treatments. (You can download our presentation here: http://www.partneringforcures.org/2010_innovators/18_findAKUre.html).
The conference, organised by the Faster Cures action tank, was impressive. It [...]
Last week was the first World Orphan Drug Congress, held in Geneva. It brought together hundreds of participants from across the industry. I attended as one of the few representatives of a patient group. I was hugely impressed. It was inspiring to see how more and more companies – including big pharma – are entering [...]
