Utrecht researchers hope for ALS breakthrough
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An experimental treatment by researchers and staff at UMC Utrecht offers hope for a small group of ALS patients. The treatment could potentially stop the deadly disease.
In the treatment patients are given an injection to prevent a defective piece of DNA affecting the body. About 7 percent of patients suffer from this form of ALS. With them, the abnormality is often hereditary. It affects about a hundred people a year.
Professor of Neurology Leonard van den Berg thinks there is a light at the end of a dark tunnel and has high expectations, though an equally hopeful study in 2011 did not lead to the desired result.
The researchers are still awaiting permission from their national body to broaden their research, but Van den Berg expects to be able to start the research before the end of the year.
The technique has already been successfully used in young children suffering from the muscle disease SMA. In addition, the disease was not only stopped, but also improved the condition of the patients. Van den Berg expects the same effect among the participating ALS patients.