Moviebox pro APK 2021 Minecraft PE APK


Collaboration is key for rare disease research. Collaboration between multiple stakeholders brings together a wide range of perspectives and experiences, leading to more effective and appropriate research. This is something we aim to promote in all of our work, and is the theme behind our new project: crowdfunding for rare disease research.

We have teamed up with the Children’s Hyperinsulinism Charity and Great Ormond Street Hospital for a new crowdfunding campaign to find a cure for the ultra-rare disease congenital hyperinsulinism (CHI).

CHI affects children from birth by causing their pancreas to overproduce insulin. As blood sugar levels drop to near zero, the brain is starved of its only fuel. This leads to seizures and lifelessness, but if it is not diagnosed early enough, it can lead to brain injury or even prove fatal. While there are a few drugs available that try and stabilise sugar levels, for a subset of children with diffuse CHI, the only treatment option is to have their pancreas removed. While this solves the CHI problem, it leaves them with lifelong diabetes. Not much of a treatment…

However, research undertaken by our crowdfunding partners may have found an alternative treatment. A repurposed drug (currently being used in organ transplant patients) has shown promise to halting CHI in children, and could even allow the children to grow out of the condition.

As dedicated Findacure fans, you will know we get quite excited about drug repurposing, especially when it can potentially save thousands of lives (and pancreases). We are therefore looking at CHI as the first condition to put through our social impact bond programme, aiming to run clinical trials into this repurposed drug.

And so we have teamed up with the CHI patient group, the Children’s Hyperinsulinism Charity, and the research team at Great Ormond Street Hospital for a crowdfunding campaign. With the funds raised, we will be able to conduct further research into the underlying mechanisms of the repurposed drug’s interaction with CHI, to prepare for clinical trials, and to run a family conference for all affected families in the UK.

Please help us by donating to and sharing the campaign with your friends, family and colleagues. Together, we can help fund a cure for thousands of children.

[/two-third][one-third]Hyperinsulinism-Photoshop-3blog 1[/one-third][/row]

Fund-a-cure for CHI

by Libbie Read time to read: 2 min