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 rare diseases, visit our What are rare diseases? page now.
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.
If you’d like to engage with the rare community to discuss the power and potential of drug repurposing, attend and sponsor our Drug Repurposing Conference. Visit our current drug repurposing conference mini site now to learn more!
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 engage with the rare community on a national and international level, attend and sponsor one of our Rare Disease Showcases. Visit our current showcase mini site now to learn more!
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.
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 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 that’s dedicated to supporting medical research charities in saving and improving lives through research and innovation. They ensure that their member charities fund the best research by developing guides, providing training and carrying out an audit of their funding processes. Learn more on AMRC’s website!
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!
Healx
Healx is a mission-driven technology company pioneering the next generation of drug discovery in order to bring novel, effective treatments to rare disease patients around the world. By combining frontier AI technology with deep drug discovery and development expertise, Healx can accelerate the pace, increase the scale and improve the chance of success of rare disease treatment development in order to meet this huge unmet need and have unprecedented patient impact. Visit Healx’s website to learn more!
Aparito
Patient monitoring made different: Mobile applications and wearable devices to monitor rare diseases. Aparito streamlines the drug development process by integrating specialist clinical and regulatory knowledge with technology for gathering patient-generated data outside of hospital. Visit Aparito’s website to learn more!
Pulse Infoframe
In 2011, Dr. Femida Gwadry-Sridhar founded Pulse Infoframe to fill a research gap that neglected millions: an inability to collect standardized, useful real world evidence in rare disease, cancer, and chronic condition populations. To increase the usefulness of this data, researchers needed to combine it with more common forms of data, such as medical reports. Collecting such sensitive data also meant developing a platform that would follow stringent regulatory guidelines, that would make it useful to researchers and advocates around the world. Thus, Pulse Infoframe was born! They’ve developed a platform to help the rare community collect and share its data and lived experiences. Real world evidence, medical records, insurance payments, trials – it’s all combined into a powerful resource to accelerate research. Visit Pulse Infoframe’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.
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 rare diseases, visit our What are rare diseases? page now.
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.
If you’d like to engage with the rare community to discuss the power and potential of drug repurposing, attend and sponsor our Drug Repurposing Conference. Visit our current drug repurposing conference mini site now to learn more!
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 engage with the rare community on a national and international level, attend and sponsor one of our Rare Disease Showcases. Visit our current showcase mini site now to learn more!
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.
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 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 University of Sheffield Advanced Manufacturing Research Centre (AMRC)
The University of Sheffield Advanced Manufacturing Research Centre (AMRC) is a network of world-leading research and innovation centres working with manufacturing companies of any size from around the globe. 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!
Healx
Healx is a mission-driven technology company pioneering the next generation of drug discovery in order to bring novel, effective treatments to rare disease patients around the world. By combining frontier AI technology with deep drug discovery and development expertise, Healx can accelerate the pace, increase the scale and improve the chance of success of rare disease treatment development in order to meet this huge unmet need and have unprecedented patient impact. Visit Healx’s website to learn more!
Aparito
Patient monitoring made different: Mobile applications and wearable devices to monitor rare diseases. Aparito streamlines the drug development process by integrating specialist clinical and regulatory knowledge with technology for gathering patient-generated data outside of hospital. Visit Aparito’s website to learn more!
Pulse Infoframe
In 2011, Dr. Femida Gwadry-Sridhar founded Pulse Infoframe to fill a research gap that neglected millions: an inability to collect standardized, useful real world evidence in rare disease, cancer, and chronic condition populations. To increase the usefulness of this data, researchers needed to combine it with more common forms of data, such as medical reports. Collecting such sensitive data also meant developing a platform that would follow stringent regulatory guidelines, that would make it useful to researchers and advocates around the world. Thus, Pulse Infoframe was born! They’ve developed a platform to help the rare community collect and share its data and lived experiences. Real world evidence, medical records, insurance payments, trials – it’s all combined into a powerful resource to accelerate research. Visit Pulse Infoframe’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.