Rare Diseases

Living With ALS: Aaron Lazar on Purpose, Creativity, and Resilience

Prenatal Therapy Approach Offers More Hope for Babies With SMA

AI and Rare Disease: Turning Insight Into Impact

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Advancing Therapy Innovation for Rare Disease Patients

Oxford-Harrington Rare Disease Centre — Turning Breakthrough Science Into Life-Changing Medicines

Hope for Duchenne Muscular Dystrophy Patients

Finding Strength in Challenges: Atticus Shaffer on Life With Osteogenesis Imperfecta

From childhood stardom to adult advocacy, actor Atticus Shaffer opens up about navigating Hollywood and life with osteogenesis imperfecta.

Prader-Willi Syndrome (PWS): A Rare Condition With Everyday Challenges

Why Early Cancer Detection Matters for Kids Like Rowan

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Access Challenges and Solutions for Rare Disease Patients

The Hidden Cost of Delayed Primary Immunodeficiency Diagnosis

Rethinking How Rare Disease Treatments Get to Patients

Life After Breakthrough Treatments: Supporting People Living With Spinal Muscular Atrophy

Once the leading genetic cause of infant death, SMA is no longer a death sentence, but living longer has introduced new challenges that must now be addressed.

The Hidden Struggle Pediatric Cancer Families Shouldn’t Have to Endure

Advancing Newborn Screening for Early Detection of Genetic Conditions

From Diagnosis to Treatment: How Rare Disease Families Navigate the Path to Genetic Therapy

Retinoblastoma: Early Detection to Save Sight

Don’t Ignore the Signs: Rare Diseases Are More Common Than You Think

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Global Genes CEO Talks Next Generation Advocacy

Next gen advocates work in many ways to accelerate progress for their disorders — and their work spans diagnosis, becoming disease experts, starting a non-profit organization, all the way to finding funders and researchers, doing research, and working with pharmaceutical companies.

Rare Disease Research Needs More Than Good Science. It Needs Bold Investment.

Protecting Innovation for Rare Disease Treatments for Patients and Families in Need

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Disability Empowerment

How Brooke Eby Is Sharing Her ALS Story

So, You’ve Received a Rare Disease Diagnosis: Now What?

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Unlocking Answers: Transforming Rare Disease Diagnosis