This year’s conference theme is “Duchenne Care: 5 Years After the 2018 Care Considerations (Lancet)”, and will take place online on June 7 and 8, 2023, from 15:00 – 18:00 CEST. Particularly, the conference will include new publications, information about the implementation of these Care Considerations in different countries. Top experts in the field will […]
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On May 2 and 3, 2023, the FDA’s Center for Drug Evaluation and Research (CDER) and the Johns Hopkins University’s Center of Excellence in Regulatory Science and Innovation (JHU CERSI) will host a jointly sponsored virtual workshop on addressing challenges in the design and analysis of rare disease clinical trials. This workshop will discuss: How […]
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The World Health Organization (WHO) is seeking experts to serve as members of the Strategic Advisory Group of Experts on In Vitro Diagnostics (SAGE IVD). This “Call for Experts” provides information about the advisory group in question, the expert profiles being sought, the process to express interest, and the process of selection. For more information […]
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As part of the NIH’s Somatic Cell Genome Editing (SCGE) program, the Office of Strategic Coordination (Common Fund) intends to publish a prize competition announcement to solicit entries for the multi-phase TARGETED (Targeted Genome Editor Delivery) Challenge. The TARGETED Challenge aims to improve the current state of in vivo delivery technologies for genome editors in two […]
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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 […]
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Prof. Guillaume Canaud (Necker Hospital for Children, AP-HP, France) offered a great presentation on “Targeted therapy for patients with PIK3CA-related overgrowth spectrum“ at the RE(ACT) Congress in Berlin (Germany). Check out his impressions on the IRDiRC Conference x RE(ACT) Congress at the following link: here.
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We empowered a community that deserves to be heard. #RAREvolution Berlin, Germany – 24 March 2023. The seventh edition of the RE(ACT) Congress and the fifth edition of the IRDiRC Conference were held jointly in-person in Berlin (Germany) from 15-18 March 2023. The event was organized by the BLACKSWAN Foundation in collaboration with the International […]
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The European Partnership on transforming health and care systems (THCS), a Cofund action under the Horizon Europe Programme designed to support coordinated national and regional research and innovation programmes along with capacity building, networking, dissemination and other key activities to support health and care systems transformation, will launch its first Joint Transnational Call (entitled “Healthcare […]
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Solve-RD, a research project funded by the European Commission for five years (2018-2022), having the ambitious goals set out by the International Rare Diseases Research Consortium (IRDiRC) to deliver diagnostic tests for most rare diseases by 2020, invites all its partners to attend the Solve-RD Final Meeting 2023 in Prague (Czech Republic) or online. The […]
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Save the date for the 9th Rare Disease Summer School organized by ITINERARE, a recently established University Research Priority Program of the University of Zurich focusing on “Innovative Therapies in Rare Diseases”. The Summer School will take place at the Kartause Ittingen, Warth (Canton Thurgau) close to Zurich from July 4th to 7th, 2023. The program includes lectures by national and international rare disease experts on topics […]
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