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

27 Feb 2026

Impact of Innovative Medicines/Health Initatives projects on rare diseases

Rare diseases face fundamental research and implementation challenges driven by small, scattered patient populations, incomplete natural history data, limited trial methodologies adapted to ultra-rare...
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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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