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...
Read more
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...
Read more
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...
Read more