The National Institute of Neurological Disorders and Stroke (NINDS) recently created the Ultra-Rare Gene-Based Therapy (URGenT) Network. The URGenT Network supports the development of state-of-the-art gene-based therapies for ultra-rare neurological diseases, […]
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Rare Diseases International (RDI) would like to thank all who attended the RDI Informal Side-Event to the 75th World Health Assembly, which took place in Geneva, Switzerland, on May 24th, 2022. […]
Read MoreIRDiRC is pleased to announce that the recommendations from the Working Group on Goal 3: Developing methodologies to assess the impact of diagnoses and therapies on rare disease patients have been […]
Read MoreIRDiRC is pleased to announce that the next edition of the 5th IRDiRC Conference and 7th RE(ACT) Congress will be held in person in Berlin, Germany from March 15th – 18th, 2023. The joint event “IRDiRC Conference […]
Read MoreRare Diseases International (RDI) is partnering with the EURORDIS Rare Barometer Survey project to launch the Global Survey on the Journey to Diagnosis for Persons Living with a Rare Disease. […]
Read MoreThe National Institutes of Health (NIH), National Center for Advancing Translational Sciences (NCATS) and the U.S. Food and Drug Administration (FDA), Center for Drug Evaluation and Research (CDER) are hosting a jointly sponsored virtual workshop to […]
Read MoreThe National Institutes of Health (NIH), National Center for Advancing Translational Sciences (NCATS) and the U.S. Food and Drug Administration (FDA), Center for Drug Evaluation and Research (CDER) are hosting a jointly sponsored virtual workshop to […]
Read MoreRare Diseases South Africa (RDSA) has a vision to see a South Africa where those impacted by rare diseases and congenital disorders have access to life-saving therapies and support for […]
Read MoreRare Diseases South Africa (RDSA) has a vision to see a South Africa where those impacted by rare diseases and congenital disorders have access to life-saving therapies and support for […]
Read MoreThe Indian Organization for Rare Diseases (IORD) has undertaken several initiatives recently to enhance the public profile of rare diseases in India. On the occasion of Rare Disease Day 2022 […]
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