Rare Disease Day 2023

 Rare Disease Day is an annual international awareness day that takes place on the last day of February. The day is dedicated to raising awareness about rare diseases and the challenges that people living with rare diseases face.

According to the National Institutes of Health (NIH), a rare disease is defined as a condition that affects fewer than 200,000 people in the United States. There are more than 7,000 rare diseases, and together, they affect more than 400 million people worldwide - our amazing Dorothy being one of them.

One of the main challenges that people living with rare diseases face is the lack of available treatments. Many rare diseases have no known cure and few, if any, FDA-approved treatments. This can be frustrating and devastating for patients and their families, who may feel like they have nowhere to turn.

Another challenge is the difficulty in getting a diagnosis. Many rare diseases have symptoms that are similar to more common conditions, which can lead to misdiagnosis or a delay in diagnosis. This can be frustrating and stressful for patients and their families, who may feel like they are being brushed off or not being taken seriously.

However, there is hope on the horizon. The NIH has established the Rare Diseases Clinical Research Network (RDCRN), which brings together researchers, patient advocacy groups, and other stakeholders to advance research and treatment for rare diseases. In addition, the Orphan Drug Act of 1983 provides incentives for pharmaceutical companies to develop treatments for rare diseases.

Despite these efforts, much more needs to be done to increase awareness about rare diseases and to ensure that people living with rare diseases have access to the care and support they need. Rare Disease Day is an important opportunity to raise awareness about these issues and to show solidarity with those affected by rare diseases.

If you or someone you know is living with a rare disease, know that you are not alone. There are support groups and patient advocacy organizations that can provide information, resources, and support. And if you want to get involved in the fight against rare diseases, consider volunteering with a patient advocacy group, making a donation to a rare disease research organization, or raising awareness about Rare Disease Day on social media or in your community. Every little bit helps, and together, we can make a difference.

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