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Oral Antisense Drug May Benefit Myasthenia Gravis

Improvements in muscle function suggest similar drugs could treat other neurological conditions

TUESDAY, Aug. 14 (HealthDay News) -- An oral antisense drug reduced muscle weakness in a small, open-label trial of patients with myasthenia gravis, researchers report in the Aug. 14 issue of Neurology. Oral EN101 antisense is a drug that inhibits the production of acetylcholinesterase.

Zohar Argov, M.D., of the Hadassah-Hebrew University Medical Center in Jerusalem, Israel, and colleagues studied 16 patients who received a four-day course of oral EN101 antisense in a phase 1b trial. Four patients subsequently received a month-long course of the drug.

The researchers found that oral EN101 antisense treatment was associated with an average 46 percent reduction in disease severity. They found that the primary benefits were improved muscle function and swallowing time, and the disappearance of a drooping eyelid. The primary side effects were mouth and eye dryness.

"Human trials of antisense oligonucleotide technology have been disappointing, but this trial suggests a therapeutic benefit, which should bolster hope for this approach for drug development," states the author of an accompanying editorial.

The study was supported by Ester Neuroscience Ltd.

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