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

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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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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