The Food and Drug Administration’s (FDA) Office of Orphan Products Development (OOPD) is pleased to announce availability of funds to support clinical trials for rare diseases and conditions. The purpose of this funding opportunity announcement is to fund clinical trials of products evaluating efficacy and/or safety in support of a new indication or change in…
The Council for International Organizations of Medical Sciences (CIOMS) has recently released a glossary of terms and definitions taken from the publicly available guidelines of the International Council for Harmonisation of Technical Requirements for Pharmaceuticals for Human Use (ICH), as a service to their followers and the ICH stakeholders, among other parties. The ICH Quality,…
IRDiRC is delighted to share the release of Connecting the dots: Embedding progress on rare disease into healthcare, an Economist Impact report sponsored by Takeda, a company that is part of IRDiRC’s Companies Constituent Committee. The report, to which IRDiRC members Takeya Adachi, Gareth Baynam and Hugh Dawkins contribute as an expert panel, takes a…
The World Duchenne Organization is pleased to announce that the WDO Leadership Award, the highest recognition for individual achievement, was awarded to Professor Francesco Muntoni on September 7, 2022. Prof Muntoni is Director of the Dubowitz Neuromuscular Centre at Great Ormond Street Institute of Child Health and Great Ormond Street hospital. This award acknowledges and celebrates…
EURORDIS is delighted to announce that applications for the 2023 edition of the EURORDIS Open Academy Schools are now open. The 2023 edition of the Open Academy Schools will take place in-person from 19-23 June in Barcelona and, following a review of our training offerings, we are excited to announce a new format for our…
The 4th edition of the Neuromuscular Translational School organized by EURO-NMD and TREAT-NMD will take place in autumn this year from November 21-25 in Leiden University Medical Center, in the Netherlands. The Translational School focuses on therapy development from bench to bedside with a focus on neuromuscular disorders. It contains lectures about the current state…
Rare Diseases International is delighted to announce their upcoming webinar on the use of the International Classification of Diseases 11th Revision (ICD -11) for rare diseases, which will take place on September 27th, at 14.00 CET. The webinar will be co-hosted by RDI and the World Health Organization. The ICD is developed and updated by the…
The Agence Nationale de la Recherche is happy to announce their upcoming ICPerMed Conference, titled « Prelude to the Future of Medicine ». The two-day conference will be held on October 5th and 6th in Paris, France. The conference will provide insight into what the future of medicine will look like by presenting concrete examples of personalised…
ERN eUROGEN is happy to announce their upcoming webinar, titled « Proximal Hypospadias: Challenges in Surgical Management ». Surgical management of proximal hypospadias is associated with major challenges regarding the establishment of a clear and effective strategy in order to optimize the functional and cosmetic outcome, reduce morbidity due to repeat interventions and minimize postoperative complications. This…
EURORDIS is pleased to announce that nominations for the EURORDIS Black Pearl Awards are now open until September, 4th 2022. The Awards are presented to patient advocates and organisations, policy makers, scientists, companies, and media in recognition of their exceptional achievements and outstanding commitment to the rare disease cause. NOMINATIONS: Just like every year, nominations can be submitted for any individual, organisation or company (including yourself…