Not everyone is given equal opportunities in life, but even when faced with the most difficult challenges, it’s not the circumstances that define us, but our outlook. Logan was born in 2007 alongside ...
KNOXVILLE, Tenn. — Bryan and Blair Begbie said all they want for Rowan — their youngest daughter — is the chance to be a kid. However, the Food and Drug Administration issued what is called a Complete ...
Runners lace up to raise money, awareness as Sachse 10-year-old battles rare, fatal genetic syndrome
There are still moments when life feels normal inside of the Sink home. But for John and Katharine Sink, not a day goes by that they don’t think of the diagnosis doctors gave 10-year-old Declan six ...
Holding back tears, Christine Ulrich recalls how her daughter Veda once loved dancing, drawing and babbling. Now, she can no longer speak or even hold a crayon. Three years ago, Veda was diagnosed ...
Ultragenyx announced significant cognitive improvements in Sanfilippo syndrome type A patients treated with UX111 gene therapy, presented at WORLDSymposium™ 2025. Ultragenyx Pharmaceutical Inc.
COLUMBUS, Ohio — Growing up, Clark Willmore always dealt with a curved back and was "a little clumsy," according to his dad Japeth. His family decided to take Clark to the doctor to learn more about ...
The microcap biotech stock had its best day ever after the FDA granted its rare-disease drug a breakthrough designation.
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