Rare Diseases 2025

Opportunities to improve the lives of those with rare conditions

The rare disease community — why it’s more than you can imagine

Rare Diseases 2025

From understanding to impact: driving academic research to deliver rare therapies

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Rare Diseases 2025

More than you can imagine: rare diseases in focus

Rare voices, bold advances: how MPN patients are helping shape medicine

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A brighter future for children with cancer

Why patient voices can help unlock rare disease treatment breakthroughs

To make a real difference in the rare disease space, pharma companies are driving innovation via partnerships with each other — and with the patients they are aiming to help. Pharma companies working in the area of rare diseases will, by definition, only impact a smaller percentage of the population. Yet, the size of a … Continued

AI-powered drug discovery company in phase 2 clinical trial for NF1 treatment

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Rare Diseases 2025

Investigating genomic newborn screening for rare conditions

Children’s charity campaigns for awareness and education of NF1

Rare Diseases 2025

Rare diseases and health equity: shifting the paradigm through global action

People, medical team and laptop for collaboration, advice and discussion for surgery or patient records. Doctor, technology and professional opinion for help, results and health in clinic or hospital

Rare Diseases 2025

Clinical clues pointing to rare disease

Rare Diseases 2025

Gain insights from the largest orphan drug and rare disease event globally

Join over 2,000 attendees, gain insights from 250+ orphan drug and rare disease industry leaders and connect with over 130 exhibitors at this event. The World Orphan Drug Congress Europe is the largest and most established event globally for orphan drugs and rare diseases. Experts and industry access The event brings together experts from across … Continued

Shining a light on rare cancers: the role of continuing medical education

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Primary biliary cholangitis symptoms, support and campaign for better care

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Protected: Joining the dots for patients living with rare primary immunodeficiencies

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Integrating patient insights into rare disease research drives meaningful change

Protected: Redefining the rare disease launch to reach every patient

Protected: The role of rare disease education in the diagnostic odyssey

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Protected: Truth and trust go hand in hand in rare disease information

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Protected: Connecting, cycling and changing children’s lives

Rare Diseases 2025

Protected: Reality series star Andy Ryan reveals rare KTWS diagnosis

Rare Diseases 2025

Protected: The vision for a competitive Europe that rare diseases offer

Vibrant blood test tubes are arranged neatly in a sleek holder, showcasing their unique colors and caps, symbolizing the critical role of laboratory testing in health assessments and diagnostics

Rare Diseases 2025

Protected: Diagnosing the world’s forgotten children through genomic testing