The 1st International Conference on Rare Diseases was held on the 1st and 2nd of March online. The event was a great success and was attended by more than 650 participants from 37 countries around the world (Europe, USA and Asia) including health professionals, researchers, academics, policy makers, industry representatives and patients. For the first…
VISION-DMD published a white paper on how to return individual clinical trial results back to patients. It focuses on addressing the ethical and technical challenges, using the vamorolone clinical trial experience. In short: Often, individual patient data is not returned to the participants, even when this is requested. ReveraGen received a grant to develop an…
EMA has recommended granting a marketing authorisation in the European Union for the first treatment that can be given orally to patients with certain types of spinal muscular atrophy (SMA), a rare and often fatal genetic disease that causes muscle weakness and progressive loss of movement. Evrysdi is indicated for the treatment of 5q SMA…
(Pre-proposal deadline: 31 March 2021) Angelini Pharma is an international group, leader in health care and well-being, committed to help patients, doctors and caregivers in the fight against diseases with constant and prevalent attention in the areas of Pain and Inflammation and Mental Health. Angelini Pharma has made research its core business to improve patients’…
The virtual event Unlocking innovation and access for rare disease patients in Europe was held online on the 22nd of February. With over 700 registrations from across the European rare disease community, the event was full of insights on the impact that policy decisions have the quality of lives of people affected by rare diseases.…
Heritable human genome editing has been proposed as a means of helping parents avoid passing genetic diseases to future generations. But can heritable human genome editing be used safely? On February 26, 2021 at 9:00 am EST (2 pm GMT / 3pm CET / 10 pm CST), join members of the International Commission on the…
The International Rare Diseases Research Consortium (IRDiRC) is glad to announce the opening of two new Task Forces: “Shared Molecular Etiologies Underlying Multiple Rare Diseases” aiming to address and document the existing challenges in adapting the basket trial approach used in molecularly targeted oncology clinical trials to drugs targeting shared molecular etiologies underlying multiple rare…
On the occasion of Rare Disease Day 2021, Biogen is pleased to invite you to the virtual event: “Unlocking innovation and access for rare disease patients in Europe” taking place on Monday, 22 February from 14:00 to 15:45 CET. Biogen’s event, done in collaboration with Reuters and EU40, will highlight the need for continued political…
Ring14 International yearly solicits international research projects focused on the Ring 14 or chromosome 14 related syndromes. The projects can be basic or pre-clinical, the ultimate goal is to find a cure for these conditions. The Board of Directors of Ring 14 International, is pleased to inform that the 2021 call for applications has been issued…
The European Medicines Agency (EMA) has published for public consultation a draft toolbox guidance on scientific elements and regulatory tools to support quality data packages for PRIME marketing authorisation applications. The EMA launched the PRIME scheme to enhance support for the development of medicines that target an unmet medical need. This voluntary scheme is based…