In the context of EJP RD’s ERN Workshops, a face-to-face workshop on clinical epidemiological research for ERNs titled « Clinical Research: The Basics & Beyond » (consisting of presentations by experts in the field of epidemiology and a feedback session on the research projects of participants) is being organised by Dr. Olaf Dekkers of the Leiden University…
The World Duchenne Organization (WDO) is hosting an online Duchenne Care Conference intended for patients, family members, clinicians and others involved in the care of individuals affected by Duchenne and Becker Muscular Dystrophy. The online conference will take place over four evening sessions of 2.5 hours each on June 1–4, 2021. The conference will provide simultaneous interpretation in various…
The International Rare Diseases Research Consortium (IRDiRC) has three Scientific Committees, one each for Diagnostics, Therapies and Interdisciplinary aspects of rare diseases research. The Therapies Scientific Committee (TSC) is a multi-stakeholder, multi-disciplinary group of experts in medical research and therapy development in rare diseases. Specifically, the TSC is devoted to pursuing the therapeutic development of IRDiRC, supporting…
NCATS along with NICHD and NINDS of the National Institutes of Health (NIH) are co-hosting a virtual NIH roundtable meeting on the theme of Gene-Targeted Therapies: Early Diagnosis and Equitable Delivery. The virtual roundtable will take place over three Thursday evenings: June 3rd, 10th and 17th from 18.00 – 22.30 CET. This meeting will bring together scientists, clinicians, therapeutics…
As part of its review of the Orphan and Paediatric Regulations announced in 2020, the European Commission (EC) has launched a public consultation for stakeholders such as patient organisations, healthcare professionals and providers, researchers, academics and representatives of the pharmaceutical industry, as well as members of the general public. Interested parties are invited to share…
In February 2021, the Coordination and Support Action on Strengthening Training of Academia in Regulatory Science (CSA STARS) Consortium organized a four-day online training course entitled « The Winding Road from a Brilliant Idea to Drug Approval » aimed at providing academic researchers with essential regulatory knowledge, case examples, insights into the regulatory system, as well as direct interaction and…
The FAIRplus project, which aims to develop tools and guidelines for making life science data FAIR (Findable, Accessible, Interoperable, Reusable) is organizing a webinar entitled « Discovering the FAIR Cookbook ». The webinar will take place on May 26, 2021 from 14.00 – 15.00 CET. The event will include a presentation of the FAIR Cookbook by the resource’s developers and project coordinator…
The Neuromuscular Disorders Applied Research Group of the World Federation of Neurology is organizing its 16th Annual Congress on Neuromuscular Diseases. The fully virtual conference will take place on May 21-22 and 28-29, 2021. The detailed program is available here, with a focus on the following themes: May 21: Muscle Disorders May 22: Peripheral Neuropathies May 28: Neuromuscular…
Action for Rare Disease Empowerment (ARDEnt), a cross-sector coalition 30 UK-based experts in rare diseases, is organizing a webinar on the findings of its Making the Unseen Seen report. The webinar will take place on May 11, 2021 from 20.00 – 21.00 CET. The ARDEnt Report reveals the impact of the Covid-19 pandemic on every stage of the rare disease…
The ProDGNE consortium, funded as part of the EJP RD’s Joint Transnational Call 2020 « Pre-Clinical Research to Develop Effective Therapies for Rare Diseases », announces its ProDGNE Kick-Off Webinar. The webinar will take place on June 03, 2021 from 18.00 – 19.00 CET. The webinar will present ProDGNE, a 3-year transnational pre-clinical research project that brings together patients and researchers from…