Just like a doctor adjusts the dose of a medication to the patient’s needs, the expression of therapeutic genes, those modified in a person to treat or cure a disease via gene therapy, also needs to be maintained within a therapeutic window.
Regenxbio eyes 2026 filing for Duchenne muscular dystrophy gene therapy – Pharmaceutical Technology
DMD is a rare, progressive genetic disorder characterised by the degeneration and weakness of muscles. Credit: Raja GamerXTC via Shutterstock. RegenxBio has revealed plans to