Exclusive: US FDA cleared pricey rare disease drug over reviewer objections

The U.S. FDA approved a pricey rare disease drug in September despite findings by its data reviewers that the treatment, ...

A Common Drug Might Help Reverse Genetic Hearing Loss, Scientists Say

A newly discovered CPD gene mutation causes hearing loss, and scientists found that Viagra and arginine may help reverse it.
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Why heart cancer is almost unheard of in humans

While cancer can strike nearly every organ in the body, the heart is a rare exception. Scientists believe this resilience ...

Gene CEP76 sheds light on cause of rare ciliopathy disorders

A Northwestern Medicine-led study has identified mutations in the gene CEP76 as a new cause of ciliopathies, shedding light on a complex group of disorders that affect multiple body systems, according ...

Extremely Rare White Iberian Lynx Caught on Camera by Amateur Photographer

An amateur photographer in Spain has captured captured a rare photo of a white Iberian lynx. Researchers believe it was a ...

Imaging study shows how brains go off-track in rare childhood disorder

Researchers at the VIB-UAntwerp Center for Molecular Neurology have visualized how brain network development is altered in a ...
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Hidden DNA mutations in healthy people may reveal how cancer starts

Scientists using TwinsUK data and ultra‑precise DNA sequencing have uncovered rare DNA mutations in healthy tissue that could ...

A New Startup Wants to Edit Human Embryos

Seven years after the first gene-edited babies were revealed, biotech startup Manhattan Genomics is reviving the idea of ...