"Monogenic" diseases, triggered by mutations in just one gene, may actually be more complex than scientists thought.
Scientists at Gladstone Institutes have flipped the traditional approach to finding potential treatments for deadly diseases.
For too long, the promise of personalized therapies has been tantalizingly close, yet frustratingly out of reach for ...
The Rich family have seen first hand how much the drug can help children with a devastating Batten disease diagnosis ...
During the Boston Globe’s Rare Disease Summit, Dr. Wendy Chung, a physician-scientist and geneticist who leads the Department ...
Just days after her son was born, Julia Friar learned he had galactosemia, a rare genetic disorder that can be ...
Scientists at Gladstone Institutes have flipped the traditional approach to finding potential treatments for deadly diseases. Instead of starting with a disease and hunting for a cure, they began with ...
I thought the hardest part of this journey would be to find a cure and a treatment for my son,” Andrew Jedlicka told The Post ...
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Transcript: Rare Diseases
Transcript: Rare Diseases ...
A panel of physicians, biotech leaders and patient advocates took aim at the FDA during a Senate hearing Thursday on how the ...
The addition of PacBio to the iHope network will further expand diagnostic access worldwide DAMASCUS, MD, UNITED ...
A rare genetic disease left a Coronado teenager legally blind at 13. Now he's running track — and helping drive research for ...
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