Misdiagnosis or underdiagnosis of rare diseases in patients with diagnoses of common diseases can lead to delayed or inappropriate treatments, thereby complicating the management of both rare and ...

This story has been updated. The human immune system is our body’s primary line of defense against harmful microbes, viruses and other invaders—but that defense line can sometimes run amok and attack ...

Scientists have uncovered the genetic cause of a mysterious multi-organ disease, tracing it to faulty versions of the SPNS1 gene that derail the body’s cellular recycling system. By revealing how ...

A team of scientists at the National Institutes of Health (NIH) used gene editing to fix the mutation that causes a form of the rare Tay-Sachs disease in mice, sparking hope that the approach could ...

Summary: Scientists have developed a targeted brain cell transplant that could transform treatment for Tay-Sachs and Sandhoff diseases. By depleting diseased microglia and replacing them with healthy ...

This BMW needed a proper shakeout—and it got one. After time in storage, we hit the streets for a full shakedown session, only to find it’s filthy, loud, and in need of attention. Ozzy Osbourne’s ...

3D rendering of a single live cell acquired through holographic tomographic flow cytometry (HTFC), showing the nucleus (purple) and aggregated lysosomes (green) within the cytoplasm (pink). This ...

NOVATO, Calif., July 11, 2025 (GLOBE NEWSWIRE) -- Ultragenyx Pharmaceutical Inc. (NASDAQ: RARE), today announced that the U.S. Food and Drug Administration (FDA) has issued a Complete Response Letter ...

Rates of cardiometabolic illnesses among American adults have reached an astonishing 50%. For diabetes, cardiovascular disease, and related inflammatory conditions, the root cause often boils down to ...