A recent study published in The New England Journal of Medicine documents a promising treatment for a common type of muscular dystrophy, called Duchenne muscular dystrophy (DMD). The experimental drug, classified as “antisense," was used in a preliminary study in four boys age 10-13.
Leg Braces and Wheelchair by Age 12
Duchenne muscular dystrophy results from a mutation in a gene that codes for a muscle protein called dystophin. Boys with Duchenne muscular dystrophy usually require leg braces by the age of 10 and a wheelchair by the age of 12. Unfortunately, the condition has no treatment and usually results in death by the age of 30 from heart or breathing problems.
Antisense Drug
An antisense drug is a man-made form of RNA that interacts with the genetic code. This particular drug was able to bind to an area of the defective gene and prevent it from being integrated into the DNA of the cell. Therefore, the abnormality in the genetic code that prevented the production of normal dystrophin was blocked, allowing for the dystrophin protein to be produced.
The initial study was limited to a single injection into the leg muscle. Further larger studies are needed in which the medicine is given systemically so that all muscles throughout the body can be affected.
Although a limited study, this could be considered a breakthrough in the treatment of muscular dystrophy and similar diseases.
Source: “Local Dystrophin Restoration with Antisense Oligonucleotide PRO051,” Judith C. van Deutekom et al, The New England Journal of Medicine, vol. 357, no. 26, Dec 27, 2007.
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For more information about Duchenne's Muscular Dystrophy, see Duchenne Muscular Dystrophy Patients at Risk for Low Bone Density.
See Chronic Pain with NMD is Undertreated to learn more about chronic pain and Charcot-Marie-Tooth disease (CMT), Guillain-Barre syndrome (GBS), post-polio syndrome (PPS), or one of the muscular inflammation disorders (myositis).
