This year, gene-editing technology was customized to fix mutations in a single patient’s genes for the first time.
U.S. Health Secretary Robert F. Kennedy Jr. on Tuesday added two rare genetic disorders, Duchenne Muscular Dystrophy and Metachromatic Leukodystrophy, to the federal newborn screening list to enable ...
Expanded newborn genetic screening can diagnose hundreds of actionable conditions, allowing for earlier treatment.
This year’s most-read articles on CKD highlighted research and insights on drug approvals, guidelines, and value-based care.
Toronto Life on MSN
“My daughter was born with a disease so rare, private companies won’t fund research because it isn’t profitable”
Laura Devlin noticed that her daughter, Penny, seemed unsteady on her feet. It took her seven years to find out why ...
In November, the Trump administration resurrected a Most Favored Nation (MFN) reference pricing policy, which would tie drug ...
Researchers have created an artificial intelligence model that can identify which mutations in human proteins are most likely to cause disease, even when those mutations have never been seen before in ...
Amber Freed tells PEOPLE how she worked for years to find a treatment for her son Maxwell's SLC6A1 diagnosis, and finally got the treatment administered in September, making history in the process.
The U.S. Food and Drug Administration said on Tuesday it had approved the first gene therapy for a rare and life-threatening ...
Scientists have uncovered a surprising genetic workaround that helps cells cope with Friedreich’s ataxia, a rare and devastating disorder.
People familiar with the plan told CNN the FDA is weighing whether to place a black box warning on COVID vaccines. These ...
While the 21st century has been bumpy, it has also ushered in monumental scientific and technological breakthroughs that have ...
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