Drug delivery strategy eliminates myotonia symptoms in mice with myotonic dystrophy
New Rochelle, NY, February 21, 2013—By targeting the specific mutation that causes the hereditary neuromuscular disease myotonic dystrophy, it is possible to neutralize the mutant RNA toxicity and minimize or even eliminate the disabling symptoms of the disease. New classes of drugs called antisense oligonucleotides are being designed to achieve this. Innovative work to develop a modified antisense drug that can be administered intravenously and achieve the desired therapeutic effect is described in an article in Nucleic Acid Therapeutics, a peer-reviewed journal from Mary ...






