Is there a treatment for Duchenne muscular dystrophy?

Duchenne muscular dystrophy is a serious hereditary muscle disorder that primarily affects boys. The disease is caused by mutations in the dystrophin gene, which is essential for maintaining muscle cell structure.

There is currently no cure for Duchenne muscular dystrophy, but there are treatments available that aim to slow the progression of the disease and improve the quality of life for patients. Medications such as prednisone and deflazacort are often prescribed to slow the progression of muscle damage. These medications can improve muscle strength and function and often slow the progression of the disease.

Exon skipping is a form of gene therapy that attempts to bypass genetic mutations in the dystrophin gene, producing a shortened but functional version of the dystrophin protein. Medications such as eteplirsen (Exondys 51) are specifically designed to target certain mutations in the dystrophin gene.

Gene therapy research is promising, with scientists attempting to directly correct or replace the dystrophin gene. Although this is still largely in the research stages, there are some clinical trials underway with promising results.

Regular physical therapy helps maintain muscle function, flexibility, and prevents contractures (tightening of muscles and tendons). Use of braces, mobility devices, and respiratory support are also important aspects of treatment.

Source: https://www.sciencedirect.com/science/article/pii/S1525001616309571?via%3Dihub

Author: Prof. Dr. Dirk Devroey - Latest update: 2024-09-02 - Copyright: Clinifacts 2024