UCLA Health researchers have created a comprehensive map showing how eight different genetic mutations associated with autism spectrum disorder affect early brain development, providing new insights ...
Orphan Drug Designation applies to approximately 50,000 Patients who predominantly suffer from severe chronic pain due ...
Researchers have identified a mechanism which can explain aspects of neurodegeneration which have baffled scientists for ...
Scientists have long known that inherited neurodegenerative disorders, including Alzheimer's, Parkinson's or motor neuron ...
US FDA grants Orphan Drug Designation to Adolore BioTherapeutics’ Kv7-activating rdHSV-CA8 therapy to treat erythromelalgia: Delray Beach, Florida Friday, January 16, 2026, 17:0 ...
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 ...
Participants most often chose goals related to daily functioning, including self-management and communicating socially. They showed significant improvements in multiple areas, including sleep, ...
“Gain of function” (GoF) research refers to genetic modification that deliberately enhances the abilities of an organism — often a virus or bacterium — to study how such changes affect its function, ...
Scientists have discovered how a mitochondrial mutation rewires immune function in a model of inherited primary mitochondrial disorders, which often lead to severe disability and death. They have ...
Angiopoietin-like protein 3 (ANGPTL3) inhibits lipoprotein and endothelial lipases. ANGPTL3 loss-of-function genetic variants are associated with decreased levels of low-density lipoprotein ...
Some results have been hidden because they may be inaccessible to you
Show inaccessible results