Orphan Drug Expert Group Webinar: Addressing the unmet needs of rare disease patients

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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,…

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EC organises European Research and Innovation Days 2021

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The European Commission (EC) is organising its annual flagship Research and Innovation event, titled « European Research and Innovation Days 2021 », bringing together policymakers, researchers, entrepreneurs and the public to debate and shape the future of research and innovation in Europe and beyond. The online event will take place over two days from June 23rd – 24th from 09.00 – 18.00 CET. The event programme consists…

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Nominations open for the EURORDIS Black Pearl Awards 2022

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EURORDIS is pleased to announce that nominations for the EURORDIS Black Pearl Awards 2022 are now open. 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, you can nominate any individual or organisation from anywhere in the world (including yourself and/or your own organisation) whose…

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EFPIA Workshop: Accelerating Adoption of Complex Clinical Trials in Europe and Beyond

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The European Federation of Pharmaceutical Industries and Associations (EFPIA) is hosting a multi-stakeholder virtual workshop titled « Accelerating Adoption of Complex Clinical Trials in Europe and Beyond » aimed at developing shared solutions for the use of complex clinical trials to optimise drug development in Europe. The online event will take place over two days: October 5th from 14.00 – 19.00 CET and October 6th from 14.00…

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EMA recommends approval for first gene therapy to treat children with rare inherited neurological disease

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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…

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Findacure’s Drug Repurposing for Rare Diseases Conference 2021

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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. …

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Workshop on clinical epidemiological research for ERNs: The Basics and Beyond

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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…

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World Duchenne Organization announces Duchenne Care Conference 2021

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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…

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