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,…
The International Rare Diseases Research Consortium (IRDiRC) and the European Joint Programme on Rare Diseases (EJP RD) are jointly organizing a two-day conference on clinical research networks (CRNs) for rare diseases, which will take place on December 1 and 2. The objective of this conference is to gather experts from different continents to increase mutual knowledge…
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…
As a continuation of the funding of an EJP RD NSS call, ECRIN is holding a two-day workshop and networking event in Prague on October 4th & 5th; the event will see the gathering of investigators involved in drug repurposing academic-sponsored trials for rare diseases, as well as other rare diseases clinical trial stakeholders. To improve the existing treatments of…
EJP RD held its annual General Assembly and Consortium Meeting on 13-15 September 2022 in Porto, Portugal. The event gathered over 100 participants from various institutions across Europe and associated countries. It was an opportunity to bring back together EJP RD members to meet in-person. Collaboration, Innovation, Data, Funding & Empowerment are key for the…
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…
EJP RD is delighted to announce an upcoming webinar on « The Statistical Evaluation of Surrogate Endpoints in Clinical Trials » which will take place online, on November 18, 2022, at 16:00 CEST. The webinar will be divided in two parts; a first half of the webinar will be devoted to the in-depth review of surrogate marker…