The RARE Drug Development Symposium, hosted by Global Genes and the Orphan Disease Center of the University of Pennsylvania, equips advocates with the knowledge, skills and connections they need to advance therapy development for their communities. This year’s theme is Innovative Ideas from Next Generation Change-Makers.The Symposium is planned to take place on April 29th-May […]
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IRDiRC Functional Analysis Task Force recently convened for a dynamic 1.5-day workshop in Paris!🇫🇷 Members of the task force from all over the world came together and discussed methodologies and strategies to advance functional analysis for rare diseases. With collaborative spirit at the forefront, the task force members worked on putting together a manuscript on […]
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The Institute for Interdisciplinary Innovation in healthcare is organizing a symposium on “Gene therapy coming of age”, taking place on 28 March, 2024, at the Palace of Academies in Brussels, Belgium. The symposium will include sessions on: Recent research and clinical advances Patient perspectives Panel debate on economic and societal challenges I3H Annual Lecture “Gene […]
Read MoreIRDiRC is delighted to co-host, together with Hope for Rare Foundation and Fudan University, the Global Rare Diseases Research Symposium & The Second China Rare Disease Research and Translational Medicine Annual Conference on May 23-25 in Shanghai (China). The conference will last for 2.5 days with 21 parallel sessions and several satellite meetings. It will […]
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A roundtable of EU and national policymakers, industry experts and patients will gather together to discuss the best approach to define unmet medical needs and Belgium’s multi-criteria approach. Around 36 million Europeans suffer from rare diseases and 95% of these diseases lack a specific treatment. Moreover, medicine development is lagging due to a risky and […]
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The European Conference on Rare Diseases & Orphan products (ECRD) is recognised globally as the largest, patient-led rare disease policy event in which collaborative dialogue, learning and conversation takes place, forming the groundwork to shape goal-driven rare disease policies and allow for important and innovative discussions on a national and an international level to take place. This 12th edition will […]
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Join us for this 2-day multi-stakeholder event on 29-30 January covering the day’s most pressing ethical issues in non-trial preapproval access at CUPA Con 2024! In-person attendees will be able to take advantage of interactive workshops, various networking opportunities, a cocktail reception, and other perks. For those unable to attend in-person, tickets providing access to […]
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IRDiRC is delighted to announce that multiple representatives of the Task Force “Drug Repurposing Guidebook”, including Therapies Scientific Committee (TSC) vice chair Anneliene Jonker (Duchenne Parent Project, The Netherlands) and TSC member Marjon Pasmooij (Medicines Evaluation Board, The Netherlands), will present at the International Drug Repurposing Conference, #iDR24, co-organised by REMEDi4ALL, Beacon for Rare Diseases […]
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On December 14, CDER and Duke-Margolis will host a jointly sponsored virtual public meeting on advancing the development of therapeutics through rare disease patient community engagement. The purpose of this public meeting is to highlight and build upon existing actionable approaches for engaging patients, patient groups, and related experts when developing necessary evidence to support […]
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The Chan Zuckerberg Initiative (CZI) is expanding the Rare As One Network and a new cycle will be open next year (including for organisations outside the USA). A call for applications is now in effect. CZI has funded 2 Cycles so far, and learned how successfully patient organisations are engaging in research collaborations, generating significant […]
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