Categories: DiscoverScientists

Scientists discover rare form of ALS that strikes children

A new form of amyotrophic lateral sclerosis that affects children has been discovered by an international team of researchers.

They used advanced genetic techniques to identify 11 such cases in children who had mysterious neurological disorders.

Most cases of ALS — also known as Lou Gehrig’s disease — are diagnosed in people between the ages of 50 and 60, and it progresses so rapidly that patients typically die within three to five years.

But this newly identified form of ALS begins in childhood, progresses more slowly, and is linked with a gene called SPTLC1, which is part of the body’s fat production system.

Preliminary results suggest that genetically silencing SPTLC1 activity may combat this type of ALS, according to the authors of the study.

“ALS is a paralyzing and often fatal disease that usually affects middle-aged people. We found that a genetic form of the disease can also threaten children. Our results show for the first time that ALS can be caused by changes in the way the body metabolizes lipids,” said study co-senior author Dr. Carsten Bonnemann, a senior investigator at the U.S. National Institute of Neurological Disorders and Stroke.

“These preliminary results suggest that we may be able to use a precision gene silencing strategy to treat patients with this type of ALS. In addition, we are also exploring other ways to ste

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