Scientists have pinpointed a previously unknown cellular breakdown that triggers multiple severe genetic diseases.
A groundbreaking FDA approval offers the first-ever treatment for ultra-rare Thymidine Kinase 2 Deficiency (TK2d). KYGEVVI, a ...
Kygevvi is indicated for patients with thymidine kinase 2 deficiency whose symptoms arise by 12 years of age. The disease ...
Everyone knows that the fall brings flu season, replete with aches, pains and a new version of the vaccine. But why is cold ...
There’s no cure for hereditary fructose intolerance. But it can be managed by strictly avoiding fructose, sucrose and sorbitol. Reading labels becomes essential for daily life, as even sauces, ...
New research from Johns Hopkins Medicine shows that the enzyme biliverdin reductase A (BVRA) plays a direct protective role ...
Q3 2025 Earnings Call Transcript November 3, 2025 BioCryst Pharmaceuticals, Inc. misses on earnings expectations. Reported ...
Pretzel Therapeutics, a leader in harnessing cellular energetics to develop novel treatments for a range of conditions spanning neurological, muscle, metabolic and rare diseases, today announced that ...
For kids with the condition, day-to-day activities like going to school or playing with friends can lead to life-threatening ...
Late last year, dozens of researchers spanning thousands of miles banded together in a race to save one baby boy’s life. The result was a world first: a cutting-edge, gene-editing therapy fashioned ...
Ageing is inevitable but the rate of decline can be offset by regular exercise, says Dr Paul Taylor, author of 'The Hardiness Effect' ...
Some results have been hidden because they may be inaccessible to you
Show inaccessible results
Feedback