Final answer:
Duchenne muscular dystrophy (DMD) is a progressive muscular disease affecting mainly boys due to a lack of the protein dystrophin. Symptoms include muscle weakness, particularly in the lower limbs, leading to difficulty walking and eventual loss of mobility. Stem cell therapy is currently being researched as a potential treatment to improve symptoms and halt progression.
Step-by-step explanation:
Duchenne Muscular Dystrophy (DMD)
Duchenne muscular dystrophy (DMD) is the most common type of childhood muscular dystrophy, primarily affecting males due to it being an X-linked recessive disorder. DMD leads to a progressive weakening of the skeletal muscles, which is caused by a mutation in the gene that encodes for dystrophin, a protein that helps maintain muscle cell structure. Without sufficient dystrophin, muscle contractions can damage the sarcolemma, leading to cellular damage and muscle fiber degradation. This results in progressive muscle weakness, particularly in the lower limbs, and can make walking difficult. Eventually, individuals with DMD may require braces for walking, and as the disease progresses, they might lose the ability to walk by the early teenage years.
While there is currently no cure for muscular dystrophy, research, including stem cell treatment, offers hope. Stem cell therapy aims to repair or replace damaged muscle tissue, potentially slowing down or halting disease progression. This cutting-edge treatment is still in the experimental stages but shows initial promise for improving symptoms and quality of life for those with DMD and other forms of muscular dystrophy.