Duchenne muscular dystrophy is a genetic disorder that causes muscles to gradually weaken over time. A person with DMD will eventually lose the ability to walk and will have problems with breathing and his or her heart.  Children who have DMD make no or low amounts of a protein called dystrophin. Dystrophin acts like glue, holding muscles together by keeping the structure of muscle cells. Without it, muscles weaken over time and become unable to work properly. A simple and inexpensive blood test called creatine kinase (CK) can help make the diagnosis. If the test results are positive, you should confirm it by DNA finger printing and muscle biopsy.

Children with DMD  might:   

(i) Not be able to walk by 15 months of age

(ii) Walk with the legs apart, on the toes, or walk with the belly pointed out (also called Lordosis), or both

(iii) Fall frequently

(iv) Need help getting up from the floor with their hands in order to stand (also called Gowers manoeuvre)

(v) Have difficulty with motor skills such as running, hopping, jumping, or climbing stairs

(vi) Have larger calves than other children of the same size or age (also called pseudo-hypertrophy)

(vii) Frequently complain of having tired legs and have behavior and learning difficulties.

Treatment option: There is no 100% specific treatments that can reverse the progression of deterioration. Treatment is aimed at keeping the patient independent for as long as possible and preventing complications that result from weakness, reduced mobility, and cardiac and respiratory difficulties. Treatment may involve a combination of approaches, including physical therapy, drug therapy, and surgery. Assisted ventilation is often needed to treat respiratory muscle weakness. Obese patients with Duchenne MD may develop obstructive sleep apnea and require night-time ventilation. Patients on a ventilator may also require the use of a gastric feeding tube.

Synthetic Steroids may be prescribed to delay muscle degeneration. Steroids such as prednisone can slow the rate of muscle deterioration in Duchenne MD and help children retain strength and prolong independent walking by as much as several years. However, these medicines have side effects such as weight gain and bone fragility that can be especially troubling in children.

Natural Ayurvedic Drugs that may help delay the loss of muscle mass: Herbal preparations such as Mamsagni, Sukumar Guggal and Fulvia, either alone or in combination, are being studied to determine if they are able to increase strength and muscle mass. These nutritional supplements have been found to protect cell membranes from oxidative injury. It may also be essential for proper mitochondrial function and cellular energy production. Scientists are investigating the safety and efficacy of Ayurvedic ingredients when added to steroids as a treatment for boys with Duchenne MD.

Enhancing natural muscle repair mechanisms: Skeletal muscle has the ability to repair itself, but its regeneration and repair mechanisms are progressively depleted during the course of several types of MD. Understanding the repair mechanisms may provide new therapies to slow, and possibly stabilize, muscle degeneration. New studies are identifying points in the regeneration-repair pathways that can be targeted by either drug or gene therapy for muscle rescue. For example, researchers have shown that chronic blockade of the muscle growth inhibitor myostatin can enhance muscle repair in animal models of MD. Other study has found that increased expression of a muscle repair protein, dysferlin, can help offset muscle damage in dystrophic animals. And the strategy of enhancing natural muscle repair mechanisms with insulin-like growth factor 2 is being used in a clinical trial for myotonic dystrophy.

Cell-based therapy: The muscle cells of MD patients often lack a critical protein, such as dystrophin in Duchenne MD or sarcoglycan in the limb-girdle MDs. Scientists are exploring the possibility that the missing protein can be replaced by introducing muscle stem cells capable of making the missing protein in new muscle cells. Such new cells would be protected from the progressive degeneration characteristic of MD and potentially restore muscle function in affected persons.

The natural regenerative capacity of muscle provides possibilities for treatment of MD. Attempts to take muscle precursor cells from fathers of Duchenne patients and implant them into patients’ muscles originally failed. However, more recent studies have focused on using stem cells to try to restore missing proteins in MD patients.

Gene replacement therapy: A true cure for Duchenne, congenital, and limb-girdle MD might be obtained if the defective dystrophin gene could be replaced by a functional gene. The large size of the dystrophin gene and the early inability of gene-delivery systems (viral vectors) to target muscle have been substantive barriers to development of gene therapy for Duchenne MD.

Over the last several years, a mini-dystrophin gene (one that is small enough for a viral carrier to deliver it) has proven successful in animal models of Duchenne MD. Viral delivery systems are much better today. As a result, researchers have made important progress in delivering a dystrophin mini-gene to muscles of a mouse model of Duchenne MD.  Scientists also are using high-throughput screening (HTS) to find drugs that increase the muscle production of the protein utrophin, which is similar to dystrophin and can help compensate for its loss.

Genetic modification therapy to bypass inherited mutations: Approximately 80 percent of Duchenne MD patients have mutations in the dystrophin gene that causes it to function improperly and stop producing the dystrophin protein. By manipulating the protein synthesis process, production of a gene that “reads through” the genetic mutation that stops production can result in functional dystrophin.

Physical Therapy, Yoga & Diet Support:  These therapies can help prevent deformities, improve movement, and keep muscles as flexible and strong as possible. Options include passive stretching, postural correction, and exercise. An Ayurvedic program is developed to meet the individual patient’s needs. Therapy should begin as soon as possible following diagnosis, before there is joint or muscle tightness.

Passive stretching can increase joint flexibility and prevent contractures that restrict movement and cause loss of function. Passive stretching on children may be easier following a ayurvedic massage (TMP procedure), warm bath or shower.

Regular, moderate Yogic exercise can help MD patients maintain range of motion and muscle strength, prevent muscle atrophy, and delay the development of contractures. Persons with a weakened diaphragm can learn deep breathing exercises that are designed to keep the lungs fully expanded.

Dietary changes: Proper nutrition is essential, for overall health. Limited mobility or inactivity resulting from muscle weakness can contribute to obesity, dehydration, and constipation. A high-fiber, high-protein, low-calorie diet combined with recommended fluid intake may help. MD patients with swallowing or breathing disorders and those persons who have lost the ability to walk independently should be monitored for signs of malnutrition.

Support aids such as wheelchairs, splints and braces, other appliances, and overhead bed bars (trapezes) can help maintain mobility. Braces are used to help stretch muscles and provide support while keeping the patient ambulatory. Spinal supports can help delay scoliosis. Repeated low-frequency bursts of electrical stimulation to the thigh muscles can produce a slight increase in strength in boys with Duchenne MD.

If your child is diagnosed with DMD, there are many groups that can help you, including the Parent Project Muscular Dystrophy: http://www.parentprojectmd.org  and the Muscular Dystrophy Association: http://www.mda.org/ . For Ayurvedic treatment you can contact the Ayush Muscular Dystrophy Society (India) http://ayush-samiti.co.in

 

 

 

 

 

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