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Stem Cell Therapy for Muscular Dystrophy

Stem Cell Therapy for Muscular Dystrophy

Gene and stem cell therapy are at the cutting edge of modern medicine, offering renewed hope to patients dealing with severe medical conditions. Previously considered a stretch of the imagination, stem cell therapy has proven effective in improving the quality of life for several patients, from regenerating damaged organs to reducing symptoms of autoimmune, neurodegenerative, and, particularly, genetic illnesses, such as muscular dystrophy.


Muscular dystrophies are a group of genetic conditions that result in weakness and the gradual decline of the heart and skeletal muscles. This condition comes in different forms, with the most common – Duchenne muscular dystrophy – affecting one out of every 3,500 boys globally.

Patients diagnosed with this genetic condition usually lack a protein called dystrophin, which makes their muscles easily prone to damage. Following this damage, patients may suffer from inflammation, which subsequently causes further damage to muscle tissues.

The significant indicator of muscular dystrophy is progressive muscle weakness. However, specific symptoms may also manifest themselves depending on the type. Some common symptoms, particularly associated with Duchenne muscular dystrophy, include;

These symptoms tend to be milder and progress slowly, usually starting in a patient’s teen years. It’s also possible for symptoms to remain hidden until patients are in their mid-20s or later.


Medical practitioners have explored several treatment options for addressing muscular dystrophy. Some current treatments include supportive care and medications. Yet, while these medications are highly regarded across the healthcare sector, they possess significant side effects. Patients have reported weight gain, delay in puberty, behavioral issues, and bone fractures.

Due to the limiting nature of current treatment options, there is a need to explore other methods to curb the progress of the disease, and stem cell therapy has proven to be a promising option, especially in Duchenne muscular dystrophy, which is also regarded as muscle stem-cell disease.




Two overall strategies are involved in addressing this disease with stem cell therapy. First, medical practitioners can engage in autologous stem cell transfer, whereby stem cells from patients are genetically altered to restore the missing protein – dystrophin – and re-implanted into the patient. For the second strategy, patients are implanted with allogeneic stem cells from an individual with functional dystrophin.

Both strategies reverse skeletal muscle damage by regenerating new muscle fibers. Bone marrow cells such as HSCs and mesenchymal stem cells (MSCs) are used for this process. These cells are multipotent and capable of differentiating into other connective tissue types. They also offer anti-inflammatory and anti-apoptotic effects and can produce extracellular matrix molecules.


Stem cells are precious in muscular dystrophy research as they not only act as models for the disease but also remain a direct and effective treatment option for the condition.

Recent tests have shown that stem cells can restore dystrophin function and rejuvenate patients’ cellular environment, thereby limiting the further progression of the disease.

R3 Stem Cell offers safe and effective stem cell therapy to treat muscular dystrophy and other neurodegenerative conditions. Contact (844) GET-STEM today.

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