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What is Muscular Dystrophy?
Muscular dystrophy is a common name for a family of diseases which cause muscle tissues to denature and be destroyed, its regeneration is impeded, and the muscle strength of the patient is reduced due to genetic mutations.
There are many classes of muscular dystrophy, each having their characteristic age of onset, symptoms and genetic mutations. Yet the common characteristics among all classes of muscular dystrophy are muscular strength degradation over time and that all muscular dystrophies are caused by genetic mutations.
The symptoms of muscular dystrophy vary depending on its type. There are mainly disorders of motor ability due to a decrease in muscle strength, reduced breathing function and impaired chewing ability. Complications in eyes, ears and brain can also be observed in some cases.
DMD is the abbreviation of Duchenne Muscular Dystrophy. A disease of the category of muscular dystrophies, and is caused by mutations on dystrophin gene.
Dystrophin gene is the blueprint of dystrophin protein, which carries the function of sustaining the mechanical strength of the muscle cells. If the genetic information encoded in dystrophin gene can’t be transcribed normally, muscles of the patient will degrade to the point which the patient loses the ability to move its body. Such is the disease named DMD.
DMD is one of the most frequent muscular dystrophy across the disease family. Almost all patients are males. Mental symptoms can be observed among DMD patients, suggesting that the disease has relation to the function of the brain.
Modern state of medicine
Currently, treatment of DMD is performed by inducing the phenomenon of “exon skipping” through drugs.
“Exon skipping” skips some regions of the dystrophin gene during translation. This enables production of dystrophin protein that carries partial function, and the loss of muscles is prevented as a result.
Yet for this treatment, since it skips parts of the genetic information, the produced protein is smaller in size compared to that of a normal dystrophin protein. While this treatment may be able to sustain the life of the patient, the patient can’t live the same life as normal peers.
We strive to offer treatment not just to elongate the life
of the patient like existing exon skipping treatment does.
Tailor-made for each patient, we offer new therapeutic molecules
that haven’t ever been seen.