Myotonic Dystrophy Are we too narrow or do we need to expand the focus?

As many of you know there is substantial research now underway for drug therapies for Myotonic Dystrophy. Drug companies are interested as it is the largest population of adult form of muscular dystrophy and it is likely that patients will need to take drugs periodically to insure the condition does not come back.

The most promising of these therapies is Antisense? What is AntiSense you wonder?
Antisense oligonucleotides – short segments of genetic material designed to target specific areas of a gene or chromosome – that activated an enzyme to “chew up” toxic RNA (ribonucleic acid) could point the way to a treatment for a degenerative muscle disease called myotonic dystrophy, said researchers from Baylor College of Medicine and Isis Pharmaceuticals, Inc., in a report in the journal Proceedings of the National Academy of Sciences.

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New Novel Drug – Testing in Myotonic Dystrophy – Isis

Recently Isis Pharmaceuticals gave a talk at the myotonic dystrophy foundation conference in San Fransisco, CA. This talk including just information that had been publicly given out previously. However, one of the lead researchers in the Myotonic Dystrophy field commented that this new drug that Isis is pursuing in collaboration with Biogen could be the “Fountain of Youth Drug”. The basic mechanism of antisense technology, if aging is caused by cell clogging with these proteins that are not being released to proper area. This could be a hug huge blockbuster drug that would exceed everyone potential.

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