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male births worldwide are affected by DMD.
of all cases of muscular dystrophy in children are caused by DMD.
years old is the age at which most individuals with DMD are unable to walk.
of individuals with DMD live beyond the age of 30.
Duchenne Muscular Dystrophy (DMD) is a genetic disorder that primarily affects boys and is characterized by progressive muscle degeneration and weakness. It is caused by a mutation in the gene that produces dystrophin, a protein important for muscle function. DMD is one of the most common forms of muscular dystrophy and is usually diagnosed in early childhood. Symptoms can include difficulty running, jumping, and climbing stairs, as well as a loss of muscle mass and mobility. Over time, the disease progresses to affect other muscle groups, including the heart and respiratory system.
There is currently no cure for DMD, but there are treatments available to manage symptoms and improve quality of life. Physical therapy, braces, and assistive devices can help maintain mobility and independence. In some cases, surgery may be necessary to correct scoliosis (curvature of the spine) or to help with breathing difficulties. There are also a number of medications that can help manage symptoms and slow the progression of the disease. Despite these treatments, DMD is a rapidly progressive disease, and most individuals with DMD are unable to walk by the age of 12. The average life expectancy for individuals with DMD is 25 years, and only 5% of individuals with DMD live beyond the age of 30. Key innovative interventions such as targeted stem cells therapy have proven to be beneficial in stopping or slowing the progression of DMD and provides hope for children with the condition.
One of the key benefits of stem cells in the treatment of DMD is their ability to differentiate into muscle cells. The lack of functional dystrophin in DMD leads to muscle damage and eventual muscle fiber death. By introducing stem cells into the affected muscles, it may be possible to replace the damaged muscle fibers with new, functional cells. This, in turn, could improve muscle function and slow the progression of the disease.
Stem cells also have the ability to secrete various growth factors and cytokines that can promote tissue repair and regeneration. These growth factors could potentially stimulate the repair of damaged muscle fibers and promote the formation of new, functional muscle tissue. This would result in a reduction of the muscle weakness and degeneration associated with DMD.
Stem cells have also been shown to have immunomodulatory properties, which could be beneficial in the treatment of DMD. Inflammation is a significant contributor to muscle damage in DMD, and stem cells have the potential to modulate the immune system and reduce the levels of inflammation in the affected muscles.
The use of stem cells as a treatment for DMD offers a promising approach for the replacement of damaged muscle fibers, the promotion of tissue repair and regeneration, and the modulation of the immune system. These effects could result in improved muscle function, slowed progression of the disease, and ultimately, a better quality of life for patients with DMD.