News
18 Nov 2024
Breaking barriers in Rare Disease Research: The Rare Disease Moonshot at two years
Rare diseases, despite affecting millions worldwide, remain an underserved area in medical research. Over 7,000 rare diseases have been identified, yet fewer than 5% have available treatments. This si...
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18 Oct 2024
Virtual Event – Innovating for Impact: Shaping the Future of Rare Disease Treatment
Millions of Europeans are living with rare diseases where no treatment option exists.
How can the work from the public and private sector help in accelerating research and raise awaren...
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16 Apr 2024
Translational research needs recommendations
The Rare Diseases (RD) Moonshot was set up to boost public private collaborations in the areas where there are with no treatment options and there is no R&D going on.
The RD Moonshot part...
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29 Feb 2024
Rare Disease Moonshot supports the EURORDIS-Rare Diseases Europe’s call for an EU action plan for Rare Diseases
On the rarest of days, we, the members of the Rare Diseases Moonshot, stand together to celebrate the resilience, strength, and perseverance of those living with rare diseases. This day serves as...
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12 Feb 2024
Recording Now Available: EJP RD-EFPIA Joint Advanced Webinar on Real-World Data, Machine Learning, and Deep Analytics
We are delighted to announce the successful outcome of the EJP RD-EFPIA Joint Advanced Webinar on Real-World Data, Machine Learning, and Deep Analytics in rare diseases, which accumulated enthusiasti...
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17 Jan 2024
Training Webinar: Real-World data, Machine learning and Deep analytics in rare diseases: Regulatory grade data collection for marketing authorization submissions – what is buzz, what is realistic?
Marc Van Dijk (UCB) and Luis Pinheiro (EMA) will present views of industry and regulators on various facets of collecting, processing, and using data for research, regulatory and healthcare decisio...
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10 Jan 2024
Rare Disease Moonshot: Europe’s Public-Private Coalition to Erase the Rare Disease “White Spots”
The knowledge and infrastructure required to unlock rare disease “white spots,” diseases where there is currently no science or translational capability, call for a different strategic paradig...
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18 Dec 2023
Diagnostic research needs recommendations
The Rare Diseases (RD) Moonshot was set up to boost public private collaborations in the areas where there are with no treatment options and there is no R&D going on.
The RD Moonshot part...
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11 Jul 2023
Clinical trials research needs recommendations
The Rare Diseases (RD) Moonshot was set up to boost public private collaborations in the areas where there are with no treatment options and there is no R&D going on
The RD Moonshot partners id...
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14 Jun 2023
Rare Diseases Moonshot partners welcome the pre-publication of the draft future Innovative Health Initiative (IHI) call topic: ‘Establishing novel approaches to improve clinical trials for rare and ultra-rare diseases’
The Rare Disease (RD) Moonshot partners welcome the inclusion of the rare and ultra-rare diseases in a recent IHI call topic which aims to establish novel approaches to improve clinical trials to ulti...
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28 Feb 2023
How the Rare Disease Moonshot can unlock science for patients
A guest blog by Magda Chlebus, Executive Director Science Policy and Regulatory at EFPIA
Sometimes the most exciting areas to work in are those facing complex challenges, where the scope for making...
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12 Dec 2022
TOWARDS A RARE DISEASE MOONSHOT: SCALING UP PUBLIC PRIVATE PARTNERSHIPS TO ACCELERATE RESEARCH
If you missed the launch of the Rare Disease Moonshot, you can watch it here...
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08 Dec 2022
‘Rare Disease Moonshot’ – Scaling-up public-private partnerships to accelerate research into world’s rarest diseases
A new initiative to boost research and development into rare and paediatric diseases will launch today at the European Health Summit.
The ‘Rare Disease Moonshot’ is a commitment and collabora...
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