Muscular dystrophy is a debilitating cluster of genetic disorders that weaken the muscles to such an extent, ultimately crushing the victim’s mobility. The disorder// hampers the victim’s quality of life, while also reducing the lifespan.
Unfortunately, the cure for this disorder still remains a burning ambition for many scientists. Duchenne Muscular Dystrophy or DMD is the most notorious of muscular dystrophy disorders. Sadly, almost 100 male children are born with this crippling disorder each year.
An experimental drug has raised scientists’ hope in being able to repair the genetic aberration, so typical among victims of Duchenne Muscular Dystrophy. The drug PTC124 proved its mettle in repairing the lost muscle function during experiments conducted on rodents. Human trial for this drug is underway, though it might take a while before the results are out.
The drug sets about the repair process by enabling cells to decipher anomalies in the genetic code pertaining to a specific protein called dystrophin. This protein is absent in 15% of patients suffering Duchenne Muscular Dystrophy. In mice, the drug assisted in superseding the mutation in the dystrophin gene, which obstructs the production of the protein.
Expressing optimism over the potential of the new drug, lead researcher Dr H Lee Sweeney said, "This new class of treatment has the potential to help a large number of patients with different genetic diseases that have the same type of mutation."
Dr Marita Pohlschmidt, director of research at the Muscular Dystrophy Campaign, extremely happy with the results of this drug said, “This drug appears to have the potential to become a treatment in the longer term for some people with DMD. We look forward to the publication of the full results of these clinical trials so that we can see more clearly what the prospects and possible timescales are for treatment.”
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