Muscular DystrophyIt can be defined as a group (heterogeneous in nature) of neuromuscular disorders. It is a group of hereditary diseases. The main symptoms of the disease include progressive muscle weakness associated with muscle wasting and, in many cases, death. The life expectancy for muscular dystrophy is dependent on the degree to which a person's muscles are weakened as well as how much the heart and lungs are affected
The voluntary muscles are affected first, especially hips, pelvic area, thighs, shoulders, and calf muscles. Later this muscle weakness also occurs in the arms, neck, and other areas. Symptoms of muscular dystrophy usually appear before age 6 and may appear as early as infancy in children.
The disease can also be identified with other physical symptoms like: Awkward manner of walking, stepping, or running, Frequent falls, Fatigue, Difficulty with motor skills (running, hopping, jumping), Progressive difficulty walking, Muscle fiber deformities Eventual loss of ability to walk (usually by the age of 12), Skeletal deformities.
Patients with muscular dystrophy are at a higher risk of acquiring neurobehavioral disorders like ADHD, Autistic-Spectrum Disorders), learning disorders (dyslexia). They can also exhibit non-progressive weaknesses in specific cognitive skills (in particular short-term verbal memory), which are believed to be the result of absent or dysfunctional dystrophin in the brain.
Inspite of enormous progress in the research and studies of molecular understanding of muscular dystrophy, there is still no cure. Much of the treatment options available aim to prevent and control the onset of symptoms to maximize the quality of life, and include the following:
- Medication like Corticosteroids to increase energy and strength and defer severity of some symptoms.
- Physical activity such as swimming which is mild and non-jarring is encouraged because inactivity (such as bed rest) can worsen the muscle disease.
- Patient is also put on physical therapy to maintain muscle strength, flexibility, and function.
- Some are also advised to use orthopedic appliances (such as braces and wheelchairs) may improve mobility and the ability for self-care.
- It is also important to take up appropriate respiratory support as the disease progresses.
Cell therapy and Muscular dystrophyThere is no cure for this disorder, but with the progressive research in stem cell therapy there are, however, several different therapeutic options under investigation. Stem cell therapy seeks to address the cause of the condition, to effect repair or reversal of the disease state through the regeneration of the affected tissue.
Cell therapy is a complete treatment. A general revitalization of organs is brought about by this therapy and an improvement in psychical and physical inconveniences.
Encouraging and pioneering experiments in respect of muscular dystrophy have shown huge promise for the disorder. Experimental procedures have been designed to strengthen stem cells' ability to regenerate damaged tissues. These procedures offer new hope to sufferers of muscle-wasting diseases such as muscular dystrophy. To make this therapy a viable treatment option for patients with muscular dystrophy, further research is going on.
There are primarily two main treatment protocols, one using umbilical cord blood-derived cells and the other an autologous procedure using the patient's own abdominal fat tissue.
Cells are made free of any infectious diseases or any contamination with the aid of through processing at certified labs.
Umbilical cord Mesenchymal stems cells show capability of fusing with existing stem cells and are also known to have anti-inflammatory properties. They thus when used in the therapy contribute a normal copy of the dystrophin gene to the faulty muscle cells. Thus the cells will be able to produce normal dystrophin. Chances of rejection with Mesenchymal cells are low as they are also known to have immuno-modulatory properties.
Stem cells are injected via the intra-venous or intra-arterial route Patients have been administered stem cell therapy by doing the bone marrow aspiration & later reinjecting the stem cell intramuscularly. This therapy has shown significant improvement in hands coordination.
Patients have also been given stem cells intravenously from HLA partially matched umbilical cord blood. A granulocytic cell stimulating factor, immune-suppressants and gamma globulin were also administered in addition. As days progressed, the defected gene showed correction; also dystrophin positive muscular fibers had increased from 2.5% to 25%. The patient showed significant gain in functions in his arms and legs.
Improvement in muscle strength, posture and improvement in balance have been manifested in patients that have received treatment. Though the extent of improvement is case dependant.
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