An Expandable Prosthesis Has Helped a Boy with Osteosarcoma Gain Freedom
Ever since the first moment he can remember, eight-year-old Dominic Gamez was drawn to travel soccer. He felt strong and powerful as his legs carried him across the field;…
Ever since the first moment he can remember, eight-year-old Dominic Gamez was drawn to travel soccer. He felt strong and powerful as his legs carried him across the field;…
In the past, it was difficult to incentivize the development of treatments for rare diseases, given issues with both profitability and small patient populations. However, all people are deserving…
Whole genome sequencing is providing high-level information about new rare diseases putting pressure on drug developers to pick up the pace. Of approximately 7,000 rare diseases, most do not have…
Hello, I'm Ailbhe and along with Adam, my husband, and Sam (5) and Dara (3), we are the O'Reilly family from Dublin, Ireland. Dara has Glut1 deficiency syndrome and was…
Over 30 million people in the European Union (EU) are living with a rare disease. Yet rare disease drug development is still somewhat stifled. Orphan designation helps to incentivize drug…
The most dramatic. Season. EVER!!! If you've ever watched "The Bachelor" or "The Bachelorette," you've probably heard this refrain from the show's host, talking up the ridiculous antics that keep our eyes…