IRDiRC 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 published in the Orphanet Journal of Rare Diseases. Access the article here: https://ojrd.biomedcentral.com/articles/10.1186/s13023-022-02337-2 The Working Group characterized a set of metrics, tools and needs required […]
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 and RE(ACT) Congress 2023” aims to bring together scientific leaders, experts and young scientists from various breakthrough scientific fields to present cutting-edge research, exchange ideas, and […]
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. This survey aims to identify factors that influence the process of obtaining a diagnosis and obstacles along the journey. This is a global survey, available […]
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 explore Regulatory Fitness in Rare Disease Clinical Trials. The
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 explore Regulatory Fitness in Rare Disease Clinical Trials. The virtual workshop will take place over two days on May 16th – 17th. Academic investigators, patient groups, and small or emerging pharmaceutical and biotechnology […]
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 an improved quality of life. One of the actions which will allow that is a validated patient registry that is confidential and secure and that […]
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 an improved quality of life. One of the actions which will allow that is a validated patient registry that is confidential and secure and that […]
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 (February 28th), a press conference was called at the State Information & Public Relations Department in Hyderabad, India as a collaborative effort between IORD and […]
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 (February 28th), a press conference was called at the State Information & Public Relations Department in Hyderabad, India as a collaborative effort between IORD and […]
Read MoreEarlier this year, the
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