Resources for researchers

Welcome to our directory for researchers!

We’ve provided the following information to help you find the answers you need quickly so that you can get involved in our work and mission.

Beacon resources

If you’d like to learn more about our charity’s vision, mission and aims, visit our Who we are page.

Researching rare conditions and developing adequate therapies for them takes significant time, money and resources. Rare science must overcome many obstacles to bring a therapy to patients quickly. Learn the challenges researchers face when working in rare disease now.

Drug repurposing, or drug repositioning as it’s sometimes called, offers a quick, cheap and accessible way to develop new rare disease treatments. Repositioning drugs substantially lowers the cost of research and speeds up the drug development process, bringing therapies to patients faster. Learn more about drug repurposing now. 

The MCDS-Therapy consortium is working to develop the first therapy for the ultra-rare bone disease, metaphyseal chondrodysplasia type Schmid (MCDS). We are running a clinical trial as part of the project to test whether the drug carbamazepine can be repurposed to treat MCDS patients. Learn more about this promising research now by visiting our MCDS-Therapy page.

Drug repurposing can only move ahead if clinical trials are funded. Our rare disease drug repurposing social impact bond proposes an innovative funding source for such research. Learn more about our social impact bond now. 

If you’d like to hear first-hand accounts of what it’s really like to live with a rare disease, we highly encourage you to listen to the Rarely Heard podcast. Visit the official Rarely Heard website now to listen to the rarely heard stories of those living with a rare condition.

We are thrilled to say that we’re working alongside major players in the rare disease space who believe in our work and mission. View our supporters now and join our growing list of collaborators!

We are proud to say that our team and co-founders have published or contributed to a host of publications, articles, reports and campaigns since we began in 2012. View our published work to learn more about our expertise and passions!

External Resources

The International Rare Diseases Research Consortium (IRDiRC)


 The International Rare Diseases Research Consortium (IRDiRC) is a global collaborative initiative launched in 2011 by the European Commission and the US National Institutes of Health.

IRDiRC’s mission is to tackle rare diseases through research and accomplish the vision to enable all people living with a rare disease to receive an accurate diagnosis, care, and available therapy within one year of coming to medical attention. Visit IRDiRC’s website to learn more!

The Association of Medical Research Charities (AMRC)


The Association of Medical Research Charities (AMRC) is a membership organisation of the leading medical and health research charities in the UK. Visit AMRC’s website to learn more!


Cures Within Reach


Cures Within Reach is the philanthropic leader in drug, device and nutraceutical repurposing research, which has the potential to transform the lives of patients with unsolved diseases by delivering treatments that can be used in the near-term. They focus on funding the projects that, when successful, will catalyze follow-on funding and/or further trials and publications. They also focus on patient access to viable treatments, regardless of whether there may be a philanthropic opportunity or commercial value to treat patients with unmet medical needs. Visit Cures Within Reach’s website to learn more!


The Castleman Disease Collaborative Network (CDCN)


For an example of a successful drug repurposing project, we highly encourage you to visit the Castleman Disease Collaborative Network (CDCN) website. The Castleman Disease Collaborative Network (CDCN) is a global initiative dedicated to accelerating research and treatment for Castleman disease (CD) to improve survival for all patients with CD. They work to achieve this by facilitating collaboration among the global research community, mobilizing resources, strategically investing in high-impact research, and supporting patients and their loved ones.