People sometimes asked Tiffany Fransen if she was drunk. She wishes more people had heard of her rare disorder.
A new CRISPR approach can control genes without cutting DNA, opening a safer path for treating genetic diseases. A newly ...
The first few months of life are a very important period in terms of human development, and at this point, many of the most ...
Three start-ups are aiming to create gene-edited babies. Columnist Michael Le Page has no doubt that editing our offspring ...
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Ultragenyx completes BLA filing for GSDIa gene therapy
Ultragenyx Pharmaceutical ( ($RARE) ) just unveiled an update. On December 30, 2025, Ultragenyx Pharmaceutical announced it had completed the ...
A recent study finds that certain psychiatric disorders have considerable genetic overlap. But these genes aren’t the whole ...
The parents of 7-week-old Dhruvan Gopi were warned he might not survive the night as specialists converged around his crib at ...
Ultragenyx Pharmaceutical said on Monday that late-stage studies showed its drug for a type of genetic bone disease failed to ...
Shaun Carmichael has polycystic kidney disease. He needs a transplant, and his wife is a match to donate. CIRCLEVILLE, Ohio — ...
Ultragenyx Pharmaceutical Inc. (NASDAQ: RARE) today announced that it has completed the rolling submission of its Biologics ...
Shaun Carmichael has polycystic kidney disease. He needs a transplant, and his wife is a match to donate. CIRCLEVILLE, Ohio — ...
Cardiovascular disease remains a leading global cause of death, and we break down its pathology, risk factors, and the ...
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