Learn more about the gene linked to the evolutionary loss of human tails, and how, in rare cases, some people are still born ...

A Northwestern Medicine study has uncovered a promising new therapeutic approach for a rare genetic brain disorder, according ...

According to a study by an international team of researchers, summarized by the Oxford University Press, Apennine brown bears ...

Leveraging modular gene editors and new regulatory pathways, Aurora will develop and commercialize therapies that can be rapidly tailored to many rare variants -- Founded by CRISPR pioneers Jennifer ...

Orphan Drug Designation applies to approximately 50,000 Patients who predominantly suffer from severe chronic pain due to ...

Researchers identified a rare genetic variant that weakens MSI2 activity in blood stem cells, slowing clonal hematopoiesis ...

Scientists have traced a devastating pattern of brain cell loss in a handful of families to a single, ultra-rare mutation that sabotages one of the brain’s key protective enzymes. The discovery not ...

A vanishingly rare genetic glitch in a single enzyme can erase a newborn’s brain cells in a matter of weeks, leaving doctors with almost no way to intervene. Researchers have now traced that ...

New insights into a sophisticated process that protects sperm cells has revealed a mechanism, similar to an immune system, thwarts genetic chaos during the earliest stages of their development.

Researchers are edging closer to a universal antiviral drug, a single medicine that could treat every virus known to man.

A long-established drug with a diverse medical history is drawing new attention for its potential role in treating an ultra-rare and life-threatening genetic disorder. Early findings suggest that a me ...

A genetic mutation passed from mother to children in families affected by schizophrenia has now been shown to completely silence a brain receptor that pharmaceutical companies are racing to target ...