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Protected: Why mRNA medicines could be a rare disease breakthrough

Rare Diseases Q1 2026

Protected: The future is now: how young people are shaping the future of rare disease advocacy

Rare Diseases Q1 2026

Protected: Changing the future of healthcare through genomics

Rare Diseases Q1 2026

Protected: Youth Driving Global Rare Disease Change

Rare Diseases Q1 2026

Protected: Closing the global diagnosis gap

Rare Diseases Q1 2026

Protected: Why Clinical Trials Matter — Especially for Rare Diseases

Rare Diseases Q1 2026

Protected: Call for rare disease global action

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Rare Diseases Q1 2026

Protected: Turning rare disease data into actionable knowledge

Protected: More Than You Can Imagine: The Unseen Work That Gets Rare Disease Medicines to Patients

Rare Diseases Q1 2026

Protected: Rare disease and the undeniable force of patient advocacy

Rare Diseases Q1 2026

Protected: Supporting those who carry the weight of rare disease

Rare Diseases Q1 2026

Protected: How we’re using artificial intelligence to advance rare disease research

Two nurses using a computer in a x-ray room

The role of rare disease education in the diagnostic odyssey

For a child with a rare disease, a doctor’s continuing education can end a years-long diagnostic odyssey and unlock access to vital care. A harmful adage in diagnosis In medical school, doctors are taught, ‘When you hear hoofbeats, think horses, not zebras,’ guiding them to focus on diagnosing common conditions and to discard the possibility … Continued

Redefining the rare disease launch to reach every patient

Rare Diseases 2025

The vision for a competitive Europe that rare diseases offer

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Connecting, cycling and changing children’s lives

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

Diagnosing the world’s forgotten children through genomic testing

Rare Diseases 2025

Reality series star Andy Ryan reveals rare KTWS diagnosis

Doctor and little boy patient. Ultrasound equipment. Diagnostics. Sonography.

Rare Diseases 2025

Building the evidence for genomics supporting newborn screening

A study led by Genomics England in partnership with NHS England is looking to understand whether we can improve the ability to diagnose and treat genetic conditions from birth. Following the Generation Study’s launch in 2024, we’re seeing real impact for families living with rare conditions. Earlier diagnoses and treatment The Generation Study is looking … Continued

Truth and trust go hand in hand in rare disease information

Doctors, conversation and people in hospital, tablet and teamwork with email for treatment approval. Employees, coworking and medical with healthcare, discussion and tech with results and schedule

Rare Diseases 2025

Improving equity for rare diseases through global policy

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

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

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Integrating patient insights into rare disease research drives meaningful change