Scientists from UC Davis Center for Surgical Bioengineering, the MIND Institute and UC Berkeley’s Murthy Lab are developing ...
A rare form of diabetes affecting newborn babies and caused by a genetic disorder has been discovered by scientists in the UK ...
Two of Opus's seven gene therapy assets are in clinical trials, with additional programs expected to enter the clinic.
Experiments on an ultra-rare genetic mutation that causes neurodegeneration in children have helped uncover a new mechanism by which brain cells die. A team led by scientists at the German research ...
A Northwestern Medicine study has uncovered a promising new therapeutic approach for a rare genetic brain disorder, according ...
Scientists from the University of Exeter and Belgium's Université Libre de Bruxelles have found that a newly identified ...
A long-established drug with a diverse medical history is drawing new attention for its potential role in treating an ultra-rare and life-threatening genetic disorder. Early findings suggest that a me ...
A new study by UCLA scientists adds to the understanding of the genetic architecture of schizophrenia. Past research has shown the impact of commonly occurring genetic variants on a person's risk of ...
It began quietly, tucked away in the immune systems of a few dozen people scattered across the globe. They weren't superheroes. They didn't wear capes. But they carried something extraordinary, an ...
Kaitlyn and Gerard Norton welcomed their daughter, Madeline, into the world. Six months later, the family found itself in a unique situation, regularly traveling to another state for medical care and ...
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