Muscular Dystrophy

Muscular Dystrophy

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We just don’t cure, we care

Muscular Dystrophy is a debilitative condition that not only drastically affects a patient’s life but also takes a heavy toll on those who love and care for him. In this race against time, we at Meditourz strive to make each day of your life ring with little joys and laughter. Our revolutionizing stem cell therapy helps stabilize muscular dystrophy symptoms and slow down progression of the disease. Apart from this, rehabilitation modules conducted by our team of expert therapists lead those battling MD to an everyday life more independent and fulfilling.

Muscular Dystrophy is a group of more than 30 inherited disorders that cause the muscles to gradually weaken and break down. It is a progressive condition and usually muscular dystrophy leads to severe disability or death.

The term comes from the Greek ‘dys’ meaning difficult and ‘troph’ meaning ‘to nourish'. Muscular dystrophy can be of several types depending on which kind of muscles are affected first, the most common being Duchenne Muscular Dystrophy (over 50% of all muscular dystrophy cases are Duchenne type) that is seen in young males between ages 2 and 6. Other types of muscular dystrophy include Myotonic MD, Becker MD, Facioscapulohumeral MD, Congenital MD, Distal MD, Emery-Dreifuss MD, Limb-girdle MD and Oculopharyngeal MD.

In the US, muscular dystrophy affects 1 male child in every 3500 individuals every year.

Muscular dystrophy is a genetic disorder caused by defects in certain genes. Most muscular dystrophies are a form of X-linked disorders that mothers can pass on to their sons even as they themselves remain unaffected by it. Some forms of muscular dystrophy are not inherited; rather they occur because of a new gene abnormality or mutation. The type of dystrophy is determined by the defective gene. The gene responsible for Duchenne Muscular Dystrophy was discovered in 1986. A year later, dystrophin, the muscle protein linked with this gene was discovered. Research is still on to discover genes responsible for other forms of muscular dystrophy.

Muscular Dystrophy can be inherited in three ways:

  • Autosomal dominant inheritance: When a child receives a defective gene from either of the parents
  • Autosomal recessive inheritance: When both the parents carry and pass on the defective gene. The parents themselves are not affected by MD.
  • X-linked (or sex-linked) recessive inheritance: When the child receives the defective gene from his mother.

Common symptoms of Duchenne Muscular Dystrophy include frequent falls, problem in getting up from a lying or sitting position, difficulty running and jumping, uneven gait, walking on toes, muscles in the calf appearing larger than usual, sore and stiff muscles and difficulty in learning.

Even though there is no cure for muscular dystrophy, there is a range of treatments available to support those with physical disabilities. In most cases, all of these may have to be put to use to help the patient.

Exercise, drugs that slow down or eliminate muscle wasting like anabolic steroids and supplementation are conventionally used to relieve symptoms.

Drug Treatment Steroids can slow down muscular dystrophy especially Duchenne type as a result delaying loss of independence, muscle strength and pulmonary functions.

Corticosteroids can enhance myoblast proliferation and promote muscle regeneration. Steroids have also been known to inhibit muscle breakdown and prednisone’s immunosuppressive and anti-inflammatory properties can reduce muscle damage and necrosis.

Apart from medication, Rehabilitation Management of muscle extensibility and joint contractures goes a long way in relieving MD symptoms. Physical therapy, occupational therapy, speech therapy and other recreational therapies can be collectively used to help maintain the patient’s independence.

Gene Therapy introduces genes into affected individuals to eventually normalize protein production. This is facilitated using viral vectors, Antisense- Induced Exon Skipping or Read through Stop Codon Strategies. However, it is a daunting task as the human genetic structure and expression is extremely complex. Also, the high cost and no human trials makes it a uncertain treatment method.

Meanwhile, research is on to derive novel treatment methods to replace or repair defective genes.

Stem Cell Therapy

Latest research has shown that stem cells have the potential to regenerate and repair damaged muscle cells. Stem cells are a great treatment method for muscular dystrophy as the mechanisms that they trigger help in repairing and restructuring tissue, reduce inflammation and prevent cell death. Adult stem cells transplanted from various tissues have been found to develop in cells of the mesoderm which form the muscle cells. This helps in restoring the stem cell pool, which in turn helps the tissue repair and regenerate.

Our stem cell transplantation protocol does not only alter the conditions of a patient, but also helps to repair and stop further progression of muscle wasting that occurs in Muscular dystrophy. This has been corroborated by the MRI-MSK reports of these patients.

  • The mechanisms by which stem cells may work are
  • Differentiation and cell fusions
  • Secretion of cytokines
  • Increase oxygen supply and contribute to vascularization in the damaged area

Stem cell thus help in tissue remodeling, reducing inflammation and preventing cell death thus promoting creation of healthier cells.

Dr. Alok Sharma

Consultant Neurosurgeon

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