When you hear hoofbeats, think horses, not zebras!

rare image

This phrase is one that many doctors learn during medical school, reminding them that when diagnosing patient symptoms, they should first consider common and likely diagnoses before looking for unusual or rare ones.

The phrase is attributed to Dr. Theodore Woodward (1914–2005), a respected physician and professor at the University of Maryland.

Today, the zebra has evolved from a symbol of medical rarity into a powerful emblem for people living with rare diseases, reminding healthcare professionals of their existence. It also highlights the ongoing need to raise awareness and ensure that rare diseases are not overlooked.

Rare diseases are not actually rare. Over 300 million people worldwide live with a rare disease. If all of these individuals lived in one country, it would be the third most populous country in the world. There are more than 7,000 identified rare diseases, and this number continues to grow.

Unfortunately, half of these patients are children, and 30% do not reach their fifth birthday.

People living with rare diseases face daily challenges, including limited treatment options and access to care. That is why one of our company’s core values is the commitment to providing timely access to life-changing therapies, improving the quality of life for our young patients and their families.

As we move through 2025, we hope to expand access to even more innovative therapies for treating rare diseases!

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