Edasalonexent is currently in clinical development for the treatment of male paediatric patients with Duchenne muscular dystrophy (DMD). DMD is a rare genetic condition caused by a mistake or abnormality in a gene called dystrophin, located on the X chromosome (one of the two sex chromosomes). Chromosomes are tiny structures inside cells made from deoxyribonucleic acid (DNA) and proteins. Males have one X chromosome, while females have two X chromosomes. As males have one X chromosome, DMD is much more common in males. DMD is fatal condition with no cure. It causes progressive muscle weakness and often leads to loss of walking ability by the age of twelve, as well as problems with the heart and lungs. Current treatment options only target a particular mistake or abnormality and focus on slowing progression of the disease.
Edasalonexent is administered orally. It works by reducing the NF-κB activity, a protein that causes inflammation leading to muscle damage and prevention of muscle regeneration seen in patients with DMD. Edasalonexent is expected to reduce the muscle damage seen in DMD and enable muscle regeneration. If licensed, edasalonexent will provide a treatment option for male paediatric patients with DMD.
rAAVrh74.MHCK7.micro-dystrophin is a medicinal product in clinical development for the treatment of children aged 3 months to 7 years with Duchenne muscular dystrophy (DMD). DMD is a rare progressive neuromuscular disorder caused by a gene mutation (change). DMD is caused by an absence of dystrophin, a protein that helps keep muscle cells intact. It affects …