The International Rare Diseases Research Consortium (IRDiRC) was launched in 2011 by a joint effort between the European Commission and the US National Institutes of Health with the vision to enable, through research, all people living with a rare disease to receive an accurate diagnosis, care, and available therapy within one year of coming to […]
IHI Call Days
The Innovative Health Initiative (IHI) Program Office just pre-published the call for proposals on “Establishing novel approaches to improve clinical trials for rare and ultra-rare diseases”. Check out additional details at the following link: https://www.ihi.europa.eu/sites/default/files/uploads/Documents/Calls/FutureTopics/DraftTopic_TrialsRareDiseases_June2023.pdf Don’t miss out the recording from the information webinar: here. The topics will be formally published at the end of July […]
IHI Announces Considered Topics for Upcoming Calls for Proposals + Related event (IHI Call Days)
The Innovative Health Initiative (IHI) has revealed the potential topics for inclusion in their upcoming calls for proposals. With the aim of enabling potential applicants to begin preparations, IHI has decided to publish the draft topic texts ahead of the official call launch, scheduled for summer 2023. IHI call 4 (two-stage call) Expanding translational knowledge […]
Join IRDiRC Diagnostics Scientific Committee!
IRDiRC’s Diagnostic Scientific Committee (DSC) plays a critical role in identifying and addressing current and future bottlenecks to rare disease gene discovery and diagnosis. As we work towards improving rare disease diagnosis globally, the DSC is currently seeking to expand its membership by welcoming a rare disease diagnostic expert from Africa or Latin America and a […]
Public Symposium at the Solve-RD Final Meeting 2023: The Impact of Solve-RD on Research & Care of Rare Disease Patients!
The Public Symposium is open to the entire human genetics and rare disease community in Europe and beyond. In addition to presentations on Solve-RD achievements, we are very happy to have two keynote speakers: Ines Thiele (University of Galway) will outline the way “Towards a personalised systems biomedical approach for the diagnosis of inherited metabolic […]
Upcoming NINDS webinar
The next installment of the “Challenges in Pediatric Neuroscience Research Webinar Series”, will be on May 12th, 2023 from 2-3 PM EST. In this episode of our ongoing series, we will be speaking with Dr. Heather Hazlett, a pediatric neuropsychologist at the University of North Carolina at Chapel Hill, about remote neuropsychological assessments. This type […]
Funding Models to Support the Spectrum of Rare Disease Research and Development
Introduction The successful development of therapies for any disease requires support from early stages (basic/fundamental research), through more mature preclinical, translational, or early clinical stages, then more mature clinical stages, and finally, post-marketing studies. How different funders decide when to fund at a given stage in a treatment’s development is fairly opaque. Knowledge of the […]
FDA Rare Neurodegenerative Disease Grant Program
In addition to the Orphan Products Grants Program that the Office of Orphan Products Development (OOPD) currently administers, a new grant program was established this year by the Accelerating Access to Critical Therapies for Amyotrophic Lateral Sclerosis Act (ACT for ALS) named the FDA Rare Neurodegenerative Disease Grant Program. This new program will be administered […]
Rare Conversations – Rare Disease Policies: Pioneering the way towards a resilient ecosystem
IRDiRC is glad to partner with Alexion’s annual Rare Conversations conference, “Rare Disease Policies: Pioneering the way towards a resilient ecosystem”, that we will partner. The event will reflect on several aspects that may be included in the new legislative framework, trying to answer the question: How to create a resilient rare disease ecosystem for the […]
Rare Conversations – Rare Disease Policies: Pioneering the way towards a resilient ecosystem
IRDiRC is glad to partner with Alexion’s annual Rare Conversations conference, “Rare Disease Policies: Pioneering the way towards a resilient ecosystem”, that we will partner. The event will reflect on several aspects that may be included in the new legislative framework, trying to answer the question: How to create a resilient rare disease ecosystem for the […]