The International Rare Diseases Research Consortium (IRDiRC) has published the results of its COVID-19 survey in the Rare Disease and Orphan Drugs Journal. The ambitious goals set by IRDiRC by 2027 to fulfill the vision of providing diagnosis and treatments to rare diseases (RDs) patients within one year of coming to medical attention have been challenged […]
Read MoreThe Food and Drug Administration
Read MoreThe Food and Drug Administration’s (FDA) Office of Orphan Products Development (OOPD) is pleased to announce availability of funds for to support natural history studies for rare diseases and conditions. These studies are intended to provide acceptable data to the FDA that will substantially contribute to the approval of new products, or new indications for already marketed […]
Read MoreFollowing a sustained campaign by rare disease patient advocacy organisations such as Rare Diseases International and with the support of several Member States, the United Nations (UN) General Assembly has formally adopted on December 16th 2021 with the consensus of all 193 UN Member States the UN Resolution on Addressing the Challenges of Persons Living with a Rare Disease and their Families. This is […]
Read MoreFollowing a sustained campaign by rare disease patient advocacy organisations such as Rare Diseases International and with the support of several Member States, the United Nations (UN) General Assembly has formally adopted on December 16th 2021 with the consensus of all 193 UN Member States the UN Resolution on Addressing the Challenges of Persons Living with a Rare Disease and their Families. This is […]
Read MoreIRDiRC member InnoSkel, a pioneering biotechnology company developing transformative therapies for the unmet needs of individuals with rare bone disorders, announced on December 16, 2021 that it has been awarded an Innovation Passport under the UK Medicines and Healthcare products Regulatory Agency
Read MoreIRDiRC member InnoSkel, a pioneering biotechnology company developing transformative therapies for the unmet needs of individuals with rare bone disorders, announced on December 16, 2021 that it has been awarded an Innovation Passport under the UK Medicines and Healthcare products Regulatory Agency’s (MHRA) Innovative Licensing and Access Pathway (ILAP), to pursue accelerated patient access for […]
Read MoreThe Therapies Scientific Committee (TSC), the Interdisciplinary Scientific Committee (ISC) and the University of Twente, the Netherlands, are jointly establishing a Working Group to explore the role and value of medical devices in rare diseases. The Working Group will primarily focus on devices used for either the treatment of rare diseases, such as implants, and […]
Read MoreThe Therapies Scientific Committee (TSC), the Interdisciplinary Scientific Committee (ISC) and the University of Twente, the Netherlands, are jointly establishing a Working Group to explore the role and value of medical devices in rare diseases. The Working Group will primarily focus on devices used for either the treatment of rare diseases, such as implants, and […]
Read MoreThe Funders Constituent Committee (FCC) have set up a Task Force to identify barriers and opportunities for the use of telehealth to improve diagnosis, care, and research experiences for rare disease patients
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