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
14 Mar 2025
Rare Disease Moonshot contribution to the Call for Evidence on the EU Start-up and Scale-up Strategy
Start-ups and scale-ups play a crucial role in the development of innovative medicines in high unmet needs areas, such as rare disease, paediatric medicines or personalized medicines. To overcome regu...
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19 Feb 2025
Rare Disease Moonshot welcomes the EU Competitiveness Compass
The European Commission’s Competitiveness Compass recognizes biotechnology, life sciences, and innovation as key drivers of economic growth and competitiveness. This communication builds on previous...
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04 Feb 2025
The Rare Disease Moonshot and Together for Rare Diseases coalitions advocate for reimagining ERNs as research powerhouses with PPPs at their core
The European Commission's recent evaluation of European Reference Networks (ERNs, representing a structured network comprising more than 1,600 highly specialised healthcare units from almost 400 hospi...
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