Isis Pharmaceuticals Develops Antisense Drug for Muscular Dystrophy and ALS

Isis Pharmaceuticals in collaboration with Genzyme and researchers at the University of Rochester Medical Center have developed a new anti-sense oligonucleotide drug to treat the most common form of muscular dystrophy, degenerative myotonic dystrophy type 1. The therapy was shown to help restore muscle movement in mice suffering from myotonic dystrophy. Isis has also developed an anti-sense oligonucleotide therapy for people with ALS, called ISIS 333611. The Phase I clinical trial results for ISIS 333611 were just released in June. The conclusions from the trial were promising, suggesting that the therapy is safe to use and well-tolerated.

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