US FDA grants orphan drug designation to AlveoGene’s novel, inhaled gene therapy, AVG-002 for lethal neonatal surfactant protein B deficiency: Oxford, UK Wednesday, December 4, ...
Sep. 9, 2024 — Researchers tested a preclinical model for an experimental gene therapy designed to treat multiple sulfatase deficiency (MSD), a disorder that affects the brain, lungs ...
The study uncovers genetic factors driving early type 2 diabetes in South Asians, highlighting risks of insulin dependence and medication response challenges.