| Research & Diagnosis on DMD |
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Foreword |
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The muscular dystrophies are a heterogeneous group of
the neuromuscular disorders. Duchenne Muscular Dystrophy (DMD) is
a most common neuromuscular disease, associated with progressive detoration
of muscle function.
The DMD disease is named after the French neurologist Duchenne De
Bulogne, who described the disorder c. 150 years back.
Duchenne patients seem able to compensate for disease process until
around five years of age when they present typically with difficulties
in climbing up stairs and keeping up with their peer at play. On going
muscle damage, tissue inflammation, attempts at regeneration, progressive
muscle loss and replacement of healthy muscle with fibro-fatty tissue
and ultimately, muscles wasting with contracturs are the unfortunate
consequences of this disease.
Typically, Ducheene patients are wheelchair bound in their early teens
and usually die of cardiac or respiratory cause or inflection.
Although, DMD initiated clinical trails roughly 150 years ago, therapies
of DMD remain supportive rather than curative |
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Present Therapeutics strategies
are: |
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- Gene Therapy Approaches.
- Cell Therapy Approaches : Myoblast/ Stem cell
- Pharmacological Therapy :Use drugs/molecules : CorticoSteroids,
Maintaining Calcium homeostasis, Decreasing inflammation, Increasing
muscle strength, Gentamycin.
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The best thing would be to replace the missing protein or fix the
broken gene that cause Duchenne MD. Scientists are trying to figure
out how to do that. But there are other things that can help us right
now. For example, there are treatments that will keep our muscles
working for longer. That is why Parent Project Muscular Dystrophy
work so hard- so they can support the scientists and doctors whom
is doing everything possible to help us. By fixing the gene someday
lots of other people may help too. Because dystrophin is one of the
biggest genes scientists know about, some people say that if you can
fix our big gene, it would make fixing little genes easier. |
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Concludingly, |
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However, the concrete result still remains unsolved. Parent Project
Organization in USA, UK, Italy, France, Germany, Australia, Italy,
Nepal, Srilanka, South Africa, Brazil etc are both jointly and separately
initiating their best efforts both individually as well as jointly
for public awareness (through several measures like meeting, media
& press conference etc), research & a hopeful way out for
treatment. (Source: Parent Project MD,USA) |
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