The Share4Rare Project announces its 1st open call for patient-driven research projects targeted towards patient organisations and researchers in the field of rare diseases to apply for conducting their patient-centred studies within the platform. The call is currently open but will close on July 15th at 17:00 CET. Applicants who are selected can avail the following benefits: Facilitation of communication with patients, their carers…
The European Expert Group on Orphan Drug Incentives (OD Expert Group), in collaboration with media partner EURACTIV, is organising a webinar on how to address the unmet needs of rare disease patients by transforming the European Orphan Medicinal Products (OMP) landscape. The webinar will take place on June 11th from 14.30 – 16.00 CET. In this online event targeted towards the broad rare disease community,…
Based on evidence from a single-arm clinical trial, the European Medicines Agency (EMA) has recommended granting a marketing authorisation in the European Union for the gene therapy SKYSONA™ for the treatment of children with cerebral adrenoleukodystrophy (CALD), a severe form of a rare inherited neurological disease. SKYSONA™, developed by bluebird bio, is a one-time treatment which can…
Findacure, a UK-based nonprofit that works in the rare disease sector, is organising its annual Drug Repurposing for Rare Diseases Conference 2021, which will be a fully online event bringing together patient groups, researchers, medical professionals and industry representatives to showcase innovative repurposing projects and encourage conversation between all members of the rare disease community. …
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…
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…
The Care-for-Rare Foundation at Dr. von Hauner Children’s Hospital, Munich has launched the Care-for-Rare Science Award 2021, a grant of €50,000 aimed at providing scientists the chance to initiate a basic or clinical research project in the field of rare diseases. The call for applications is currently open and will close on July 31, 2021. The award…
Global Blood Therapeutics (GBT) is organizing a European multi-stakeholder roundtable event titled « Addressing Inequalities in Healthcare – Spotlight on Sickle Cell Disease Patients in Europe ». The webinar will take place on May 11, 2021 from 14.30 – 16.00 CET. The session will feature discussions of the health inequalities and challenges that disproportionately impact minority groups across Europe. In particular, the…
We recently worked with Mediaplanet UK on the 2019 Rare Diseases Campaign! Read the interview where Dr. Daria Julkowska (scientific coordinator of the EJP RD) speaks about how the European Joint Programme on Rare Diseases can help turbo-charge the research for the benefits of patients. situs toto bo toto situs togel situs togel bet togel…