An international team of researchers recently discovered a new form of amyotrophic lateral sclerosis or ALS affecting children.
A Medical Xpress report said the study authors used advanced genetic techniques to determine 11 cases in children who suffered mysterious neurological disorders including ALS.
Most occurrences of ALS, also known as Lou Gherig's disease, are diagnosed in people whose age ranges between 60 and 60 years old, and it progresses so fast that patients usually die within three to five years.
However, this newly detected ALS form starts in childhood, advances more slowly, and is associated with a gene identified as SPTLC1, which is part of the production system of the body.
Precision Gene Silencing Strategy
Authors of the study said, initial results of the research, Childhood amyotrophic lateral sclerosis caused by excess sphingolipid synthesis, published in Nature Medicine, propose that genetically silencing SPTLC1 may fight this type of ALS.
According to the United States National Institute of Neurological Disorder and Stroke Senior Investigator Dr. Carsten Bonnemann, ALS is a paralyzing and frequently deadly disease that typically impacts middle-aged people.
They found that a genetic form of the disorder can threaten children, as well. The study findings show for the first time that amyotrophic lateral sclerosis can result from changes in the manner the body is metabolizing lipids.
These preliminary results added Bonnemann, suggest that the precision gene silencing strategy may be used to treat amyotrophic lateral sclerosis in children.
ALS at an Early Age with Slowly Progressing Symptoms
The researchers are also exploring other approaches to step on the brake that slows the so-called SPT activity, explained Bonnemann in a news release.
Their eventual goal is for these ideas to be translated into effective treatments for the patients who presently have no therapeutic options.
Initial symptoms of ALS in the 11 children reported appeared at about four years of age. By the end of the research, this report specified, "they had lived from five to 20 years longer."
Dr. Payam Mohassel, the study's lead author and an NIH clinical research fellow said, these young patients experienced upper and lower motor neuron problems that are suggestive of ALS.
The lead author added, what made the cases unique was the onset at an early age, not to mention the symptoms' slower progression. This made the researchers wonder, what was underlying this distinctive form of ALS.
ALS Cases
The study started with a young woman namely, Claudia Digregorio, from the Apulia region of Italy, ScienceDaily reported.
Her case, this report specified, had been so disturbing that Pope Francis conveyed in an in-person blessing on her at the Vatican prior to her trip to the US to be checked by the team of Bonnemann at the NIH's Clinical Center.
Digregorio, like many of the other patients, needed a wheelchair so she could move around. She also needed a tracheostomy tube to support her breathing.
Neurological examinations by the team showed that she, as well as the others reported, had several of the signs of ALS, which include severely weakened or paralyzed muscles.
Moreover, the muscles of some patients showed signs of atrophy when examined through a microscope or non-invasive scanners.
Related information about ALS in children is shown on The ALS Association's YouTube video below:
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