Stem Cell Therapy for Muscular Dystrophy

What Causes Muscular Dystrophy?

Muscular dystrophy is a group of genetic diseases that cause progressive weakness and degeneration of skeletal and heart muscles. These are the muscles that control movement and heartbeats, respectively 1. It affects about 6 out of 100,000 children in North America and Europe. Muscular dystrophy is caused by:
  • Genetic mutations that lead to the production of abnormal or missing proteins necessary for muscle fiber function
  • Spontaneous (not inherited) gene mutations during fetal development 
There are several types of muscular dystrophy, each caused by a different gene mutation 2:
  • Duchenne muscular dystrophy (DMD): A disorder caused by a deletion in the DMD gene on the X chromosome. The DMD gene provides instruction for making dystrophin, a connective protein necessary for muscle and heart muscle functions. Because females have two X chromosomes and males have one X chromosome, DMD affects mainly boys. Symptoms can begin between ages 2 – 16, but may also show up as late as 25.
  • Becker muscular dystrophy: A muscle disorder caused by a milder change in the DMD gene than the deletion that caused Duchenne muscular dystrophy. As a result, Becker muscular dystrophy comes from reduced dystrophin, rather than complete elimination.
  • Myotonic dystrophy: A muscle disorder resulting from abnormal production of myotonin protein kinase, an enzyme involved in communication across muscle cells. Myotonic dystrophy causes muscle wasting, cataracts, and balding. 
  • Limb-girdle muscular dystrophy affects both genders and involves a variety of different gene mutations
  • Facioscapulohumeral muscular dystrophy affects the face, shoulder, and upper arm muscles
The symptoms of muscular dystrophy vary depending on the specific type of the disease and the age at which the symptoms begin. Common symptoms include:
  • Progressive muscle weakness beginning in the pelvic and shoulder muscles, and eventually affecting all other muscles in the body 
  • Cardiomyopathy, where the heart muscle becomes weak and enlarged, and can lead to death
  • Difficulty walking
  • Muscle wasting, where the muscles become weaker and smaller in size
  • Delayed motor milestones such as sitting, crawling, or walking
  • Difficulty breathing due to weakened muscles that are involved in breathing and postural issues
  • Vision and hearing loss
  • Cognitive dysfunction and delayed cognitive development, such as deficits in verbal, short term, and working memory

Stages of muscular dystrophy

Muscular dystrophy worsens over time. The rate of progression varies, but there are generally four stages of muscular dystrophy:

  1. Early-stage patients experience some muscle weakness and difficulty with certain movements, such as climbing stairs or standing up from sitting. They can still perform most activities of daily living without assistance.
  2. Ambulatory patients experience significant muscle weakness and may need to use mobility aids, such as braces, crutches, or a wheelchair, to get around. They can still walk and stand with assistance.
  3. Non-ambulatory patients cannot walk and require a wheelchair for mobility. They may also need assistance with activities like dressing and grooming.

patients need 24-hour care and assistance with all aspects of daily living. Respiratory and heart function is also severely compromised and leads to death in many cases.

Stem Cell Therapy for Muscular Dystrophy FAQ

The Scientific Rationale Behind Stem Cell Therapy for Muscular Dystrophy

Currently, there is no known cure for muscular dystrophy. The standard-of-care treatments may slow down disease progression and manage symptoms but do not reverse the damage done to the respiratory system. Most drugs for muscular dystrophy also have unpleasant side effects that tend to lower the quality of life. Some patients may not respond well to the available treatments or may not be eligible for certain treatments due to other health conditions or contraindications. 

This is where stem cell therapy can uniquely deliver benefits.

How Much Does Stem Cell Therapy Cost for Muscular Dystrophy?

The cost depends on the specific treatment you will be undergoing and how many treatment sessions you need. Book a consultation with us today to get your personalized stem cell therapy treatment plan.

How Many Treatments Will I Need?

Typically, you will need 1-2 treatments, depending on the plan of care established between you and your doctor. All treatment appointments are in person, but the initial consultation and follow-up can be done by phone.  

Some patients come back for additional treatments over the course of months or years. This largely depends on the stage of your disease, how well you respond to the treatment, and what your treatment goals are.

Your Patient Journey with Stem Cell Therapy for Muscular Dystrophy

Step 1: Application and initial consultation

Our questionnaire and initial consultation help us determine whether you’re a good candidate for stem cell therapy, along with the best treatment plans for you. Our patient advocate will walk you through the process, pricing, and payment plans.

Step 2: Physical exam

You’ll come in for a physical exam in preparation for the treatment and also to assess your current symptoms and health status.

Step 3: Stem cell collection

Your procedure will begin where we harvest cells from your bone marrow or fat tissues. We will then isolate the stem cells from these tissues in the clinic before administering your first treatment

Step 4: Stem cell culture

If applicable, we will send your stem cells to the lab to expand them in numbers. 

Step 5: Future treatments

Your future stem cell treatments will be done using your cultured stem cells. 

Step 6: Follow-up appointments

Our team will keep in contact with you to monitor your treatment progress and support you through your recovery.

Advantages of Stem Cell Therapy vs Standard Treatments and Other Alternative Treatments for Muscular Dystrophy


Regenerates new neurons instead of treating the symptoms of neuron loss.

Minimal Side Effects

Peace of mind knowing you won’t experience the downsides of traditional treatment approaches.

Deeper Approach

Treats a fundamental underlying mechanism of the disease.

Results May Last Years

By combatting neuron loss, benefits may last many months to years.

No Daily Pill To Take

Nothing to do after your in-office treatment except let your body do its job.

Harnesses Your Own Healing Power

Use the power of your own stem cells and biology to do the healing.

Current standard-of-care treatments for Muscular Dystrophy and their shortfalls

The current standards of care for muscular dystrophy include a multidisciplinary approach to address issues of mobility, strength, and pain management. Some specific interventions include:


These may include muscle relaxants,and steroids to help manage weakness and other symptoms.
  • Steroids, such as prednisone or deflazacort, slow the progression of muscular dystrophy by reducing inflammation and promoting muscle growth 16. Side effects can include:
    • Weight gain
    • Mood changes
    • Osteoporosis (weakened bones)
    • Delayed growth
    • Increased risk of infection through suppression of the immune system
  • Baclofen is a muscle relaxant that reduces stiffness and spasms. Baclofen increases the activity of the neurotransmitter GABA through the GABA-B receptor in the brain to reduce muscle activity 17. Side effects can include:
    • Drowsiness
    • Weakness
    • Nausea
  • Dantrolene reduces muscle stiffness, spasms, and creatine kinase (CK) – a marker of muscle inflammation. Dantrolene blocks the ryanodine receptor on muscle cells and prevents the release of calcium and muscle contraction 18,19 Side effects can include:
    • Drowsiness
    • Dizziness
    • Nausea
    • Diarrhea
    • Weakened breathing

Physical and occupational therapy

Physical therapy (PT) improves muscular symptoms of muscular dystrophy like 20,21:
  • Muscle strength
  • Coordination
  • Balance 
  • Posture 
  • Range of motion
  • Mobility
Use of assistive devices also helps with mobility, communication and balance. These may include 
  • Orthotics
  • Braces
  • Walkers
  • Wheelchairs
  • Communication devices
Occupational therapy helps with activities of daily living, such as 
  • Dressing
  • Bathing
  • Grooming 
Occupational therapy (OT) also provides adaptations to home or work environments to make daily activities easier and safer.  Physical and occupational therapy, however, do not address the root causes of the disease and may not slow down or reverse disease progression.

Gene therapy

Gene therapy is an emerging treatment that involves replacing or repairing the faulty gene responsible for the disease. While gene therapy shows promise as a potential cure for muscular dystrophy, it is still in the experimental stage and has not yet been approved for widespread use 22.

Meet The Doctors

Bringing years of experience in the emerging and innovative field of interventional and regenerative orthopedics to the Nashville, Tennessee area.

Dr. Ethan Kellum, M.D.

Dr. Hemal V. Mehta, M.D.

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  15. Youngest child in world gets stem cell therapy for cerebral palsy at StemRx Hospital.
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