World Awareness Day for Duchenne Muscular Dystrophy
On the occasion of World Awareness Day for Duchenne Muscular Dystrophy, the Paediatric Neurology section in Paediatrics Department, in collaboration with the Rehabilitation Centre, Public Relations and Media Affairs at National Guard Health Affairs - Jeddah, is organizing an interactive meeting for all families with Duchenne.
The aim of this awareness day is to discuss the standard of care and the latest developments, as well as strategies to create a positive environment for a better future for our children. The lectures will be accompanied by educational recreational activities for all children throughout the day.
Duchenne is caused by a genetic mutation that prevents the body from producing dystrophin, a protein that muscles need to work properly. Without dystrophin, muscle cells become damaged and weaken. Duchenne muscular dystrophy is considered as a rare muscular disease. It affects approximately 1 in 3500 live male birth. It is characterized by a rapid and relentless decline in physical function, with subsequent respiratory and cardiac failure, leading to early death.
Initial uncertainties arise because of one of the following three signs :Delay in the ability to walk; followed by difficulty in running or climbing stairs , Delay in speech and High levels of muscle or liver enzymes in the blood test .
The importance of early diagnosis of Duchenne Muscular Dystrophy will help for better opportunities for optimised care, including steroid treatment, physiotherapy and screening for cardiomyopathy, in addition Novel treatments are most successful in early stages of the condition when the muscles are less damaged.
Initial clinical studies began in late 2017 to test the safety and effectiveness of gene therapy, which replaces the damaged gene with a healthy gene, to allow the production of natural dystrophin.