Pediatric (onset before age 18) and juvenile (onset before age 25) Amyotrophic Lateral Sclerosis (ALS) is a rare, life-threatening, progressive neurodegenerative disease occurring in children, teenagers and young adults.  ALS attacks and destroys motor neurons which strips a person of their ability to move, walk, talk, eat and breathe eventually leading to death.  

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ALS knows no racial boundaries and cases of pediatric and juvenile ALS have been documented in medical literature around the globe in both males and females. Pediatric and juvenile ALS can follow a slow or aggressive disease progression dependent upon the person's specific gene mutation. 

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The majority of reported cases of pediatric and juvenile ALS are caused by a genetic mutation. Many of these mutations are found to be de novo meaning they are not inherited from either parent. The most prevalent mutated genes include SOD1 (superoxide dismutase one), SETX (senataxin), or FUS (fused in sarcoma). Several other genes have also recently been linked to this disease in children, teenagers and young adults. Current research has shown that over 50% of Pediatric ALS cases are due to FUS mutations.  Genetic testing should be pursued in young people presenting with symptoms of ALS. 

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Research of pediatric and juvenile ALS causing genes is desperately needed in order to discover treatments and eventually lead to a cure for this fatal disease.  Gene silencing therapies such as antisense oligonucleotide (ASO) and RNA interference (RNAi) along with gene-editing tools such as CRISPR/Cas-9 are showing potential as powerful and promising ALS treatments.