Experiments on an ultra-rare genetic mutation that causes neurodegeneration in children have helped uncover a new mechanism by which brain cells die. A team led by scientists at the German research ...

Scientists at Mount Sinai have created an artificial intelligence system that can predict how likely rare genetic mutations are to actually cause disease. By combining machine learning with millions ...

A review article led by researchers from the B·ARGO group at the Germans Trias i Pujol Research Institute (IGTP) and from the Medical Oncology Department of the Catalan Institute of Oncology (ICO) in ...

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Orphan Drug Designation applies to approximately 50,000 Patients who predominantly suffer from severe chronic pain due to hereditary neuronal sodium ion channel mutations Adolore's Kv7 voltage-gated ...

The US FDA approved 46 new drugs in 2025, despite a tumultuous year at the regulatory agency.

Victoria Gray spent 34 years battling the debilitating pain of sickle cell disease. Then she volunteered to be the world's first "prototype" for a CRISPR therapy, based on technology invented at UC ...

In conjunction with the strategic evolution, the Company appointed Jian Irish, Ph.D., M.B.A., as Chief Executive Officer. Based on a revised capital allocation strategy, the Company extended its ...

Learning doesn’t always mean flipping through heavy textbooks or sitting in a long lecture. Sometimes, it just takes ...

2019-06-12 A deepfake video of Mark Zuckerberg presents a new challenge for Facebook 2019-06-12 Top 2020 Democrats best Trump in hypothetical matchups in new poll 2019-06-12 Elon Musk says 'there is ...

Genetics aren't necessarily destiny for those with mutations thought to always cause inherited blindness, a new study says. Fewer than 30% of people with these genetic variants wind up blind, even ...