People sometimes asked Tiffany Fransen if she was drunk. She wishes more people had heard of her rare disorder.

Kerala doctors successfully performed a life-saving liver transplant on a two-and-a-half-year-old child diagnosed with the ...

Marci Shelman and Tristan Dederscheck’s “miracle” baby was diagnosed with neonatal Marfan syndrome. After four months in the ...

Experiments on an ultra-rare genetic mutation that causes neurodegeneration in children have helped uncover a new mechanism ...

Fakeeh University Hospital is emerging as a global destination for advanced gene therapy, delivering highly specialized ...

The treatment uses a modified, harmless virus to deliver healthy genes to a patient’s cells to treat genetic diseases. Read more at straitstimes.com. Read more at straitstimes.com.

For the first time, doctors have treated a baby born with a rare, life-threatening genetic disorder with a gene-editing therapy scientists tailored to specifically repair his unique mutation. The baby ...

U.S. Health Secretary Robert F. Kennedy Jr. on Tuesday added two rare genetic disorders, Duchenne Muscular Dystrophy and ...

This year saw notable progress in head and neck cancers, Huntington's disease, personalized genetic therapy, and heart ...

A family on Brookside’s “Candy Cane Lane” has transformed their home into a winter wonderland to raise money for their son's ...

Ultragenyx Pharmaceutical said on Monday that late-stage studies showed its drug for a type of genetic bone disease failed to ...