Booster toward new scientific breaktroughs in rare and pediatric diseases
ABOUT THE MOONSHOT
95% of the 6,000-7,000 identified rare diseases don’t have an approved therapy. For most of these diseases, research is effectively non-existent. The search for new treatments is thwarted by enormous scientific challenges. At the current pace, it would take over 100 years to develop treatments for all rare conditions.
Public-private partnerships could help pool resources to solve problems more quickly, reduce fragmentation and scale up existing initiatives to make a real difference for patients. That’s why a coalition of partners is joining forces to accelerate research in rare and paediatric diseases and bring new therapies to patients.
Latest news
06 Nov 2025
Together For Rare Disease conference outcomes on ‘Boosting EU competitiveness through PPPs in RD’ translate the Rare Disease Moonshot contributions into action for all stakeholders.
The European Union's commitment to strengthening its competitiveness agenda has created unprecedented opportunities for rare disease research and innovation. In 2025, the Rare Disease Moonshot contrib...
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19 Jun 2025
Rare Disease Moonshot’s contribution to the Consultation on the EU Biotech Act
The Rare Disease Moonshot welcomes the European Commission’s ambition to develop a comprehensive EU Biotech Act. This initiative represents a timely opportunity to unlock the full po...
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14 May 2025
Rare Disease Moonshot’s contribution to the Consultation on the next Multiannual Financial Framework
Introduction
The Rare Disease Moonshot is a multi-stakeholder initiative uniting public and private actors, researchers, clinicians, patients, and funders to advance research and care for the 30 mill...
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