Scientists discovered that certain gene changes allow cells to function even when frataxin, the protein lost in Friedreich’s ataxia, is missing. Experiments in worms, human cells, and mice revealed ...

While deadly diseases had some pretty terrible outcomes throughout history, they also have led the way for incredible ...

Lurie Children's Hospital officially announced an expansion of research with the $11 million from Don and Anne Edwards, who ...

Complicated follows several children with Ehlers-Danlos syndrome and their parents trying to get care. Not only are doctors ...

We grasp the central theme of Steven Feinartz’s documentary “Are We Good?” –– playing at the Martha’s Vineyard Film Center starting Dec. 5 –– just moments after the opening credits roll by. On May 18, ...

The December 4 showing [7-9 pm] at the Quad Cinema will include a Q&A with director Abrahams and other film subjects moderated by Schrier, who is featured in the film and has Ehlers-Danlos Syndrome ...

Instead of requiring personalized gene edits for each patient, the new approach could create a standardized method to use for many diseases. By Pam Belluck and Carl Zimmer Gene-editing therapies offer ...

Across America, millions of parents of children with rare diseases are in a race against time, hoping that new treatments will be developed fast enough to save their kids. Thanks to advances in ...

Selena Quintanilla-Pérez’s widower is looking back at his life with the Queen of Tejano music. Three decades after the “Bidi Bidi Bom Bom” singer’s tragic death at age 23, her husband Chris Pérez—whom ...

One in 5 of the highest-revenue drugs of 2022 was exclusively approved for rare conditions, accounting for more than 7% of US pharmaceutical spending. Study Design: Cross-sectional study. Methods: ...

The comedian was joined by seven of his kids — Myles, Christian, Eric, Bella, Shayne, Zola and Bria Presley Ann/Getty Eddie Murphy had his family's support as he walked the red carpet for the premiere ...