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 ...

New name reflects the Company’s strategic evolution focused on later-stage preclinical assets leveraging its most advanced, signature gene-editing capabilities ...

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 ...

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 ...

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 have developed a new artificial intelligence tool that can identify harmful genetic mutations and predict the types of diseases they are likely to cause, offering faster diagnosis and new ...

A sperm donor whose samples helped conceive nearly 200 children across Europe unknowingly carried a cancer-causing genetic mutation — a hidden risk now tied to multiple childhood illnesses and early ...

Danish researchers have found a molecular switch that lets plants partner with nitrogen-fixing bacteria instead of fighting them, opening the way to self-fertilizing cereal crops like wheat and barley ...

A single sperm donor who carries a rare cancer-causing genetic mutation has fathered at least 197 children across 14 countries in Europe, according to a collaborative investigation by 14 European news ...

Andrew Joseph covers health, medicine, and the biopharma industry in Europe. You can reach Andrew on Signal at drewqjoseph.71. LONDON — A sperm donor who unknowingly carried a gene variant that can ...

On the surface, cystic fibrosis and Tay-Sachs disease have nothing in common. Although both are inherited genetic disorders, one causes thick mucus buildup in the lungs, making it progressively harder ...

The genetic code is the recipe for life, and provides the instructions for how to make proteins, generally using just 20 amino acids. But certain groups of microbes have an expanded genetic code, in ...