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Collecting Rare Disease Data: Why it Matters

Rare diseases affect a relatively small number of people, so it can be difficult for healthcare providers, government agencies, advocacy groups, pharmaceutical companies, and others to access information about the experiences of those living with rare diseases.

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Become Part of the Solution: Participate in Clinical Research

Clinical research plays a critical part in advancing science and treatments. Volunteers who participate in this research are essential – new treatments for rare diseases cannot be approved without them.

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Genetic Testing 101: Know Your Genes

Eighty percent of rare diseases are genetic.1 Genetic testing is a tool that can help identify these conditions in individuals and families. It helps empower people to make informed decisions about their health.

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Rare & Prepared: Emergency Planning for Individuals with Complex Healthcare Needs

Preparing for an emergency is important, especially if you or someone you care for has a rare or chronic condition and/or special healthcare needs. We’ve put together a few tips on how to prepare so you’re ready for (almost) anything.

Your Voice Matters

Your Voice Matters: How to Engage with the FDA

Yes! The Food and Drug Administration (FDA) is the regulatory agency in the United States that reviews and approves (foods and) medicines for use in people. The FDA’s job is to ensure that medicines are safe and effective, and provides the public with accurate, science-based information about these medical products.

Spotlight on Rare Disease Caregivers

Caring for someone with a rare disease can be an around-the-clock commitment, leaving caregivers with little time to attend to their own needs. Forty percent (40%) of rare disease caregivers report having fair or poor emotional or mental health and thirty percent (30%) report having fair or poor physical health.

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Transitioning to Adulthood

Everyone goes through transitions, or changes, at certain times in their life. During these times your day-to-day routine, priorities, responsibilities, or disease management requirements may change. For someone living with a rare or chronic condition, one of the biggest changes is moving from pediatric to adult care.