A rare genetic disorder discovered by UT Southwestern Medical Center researchers and their colleagues can cause brain damage ...
In a breakthrough study, scientists have discovered that a variant in one gene, GRIN2A, can directly cause mental illness – ...
On a video call in early September, Sarah Tabrizi first saw the data that she and other researchers studying Huntington’s ...
Australia prides itself on fairness and opportunity, yet an unspoken pecking order shapes who advances and who is blamed for ...
Praxis Precision Medicines, Inc. (NASDAQ: PRAX), a clinical-stage biopharmaceutical company translating genetic insights into ...
This year, gene-editing technology was customized to fix mutations in a single patient’s genes for the first time.
Following a single subcutaneous 240 mg dose, WVE-007 (INHBE GalNAc-siRNA) improved body composition at three months compared ...
To date, ETX101 has shown a favorable safety profile and has been well-tolerated across all four dose levels with no treatment-related serious adverse events (n=19). The most common treatment-related ...
The EMBOLD topline results will be presented at the American Epilepsy Society Annual Meeting at the Georgia World Congress Center, Poster Hall B2, 1.38 from 12:00-2:00 p.m. on Saturday, December 6, ...
The DNA inside our cells is constantly being damaged, and one of the worst kinds of damage is a double-strand break—when both ...
Capricor Therapeutics said on Wednesday its cell therapy for a rare muscular disorder met the main goal of a late-stage study, reviving hopes for U.S. approval after a setback and sending its shares ...