pediatric ALS

Clinical Trials in Pediatric ALS ‘Simply Not Feasible,’ Scientists Argue

The rarity of amyotrophic lateral sclerosis (ALS) in children makes it impossible to realistically conduct clinical trials of experimental medicines in this population, researchers across Europe argue in a paper. “Given the absence of consensus on pediatric ALS as a disease entity, the extremely low prevalence of ALS in…

Childhood Form of ALS Identified, Linked to SPLTC1 Mutations

A previously unknown form of amyotrophic lateral sclerosis (ALS) — one with onset during childhood — is caused by mutations that alter the production of certain lipids (fat molecules), scientists report. “We found that a genetic form of the disease can also threaten children. Our results show for the first…

FUS Mutations Cause Most Cases of ALS in Adolescents, Literature Review Finds

When amyotrophic lateral sclerosis (ALS) symptoms emerge during adolescence, mutations in the FUS gene are likely to blame, findings from a case report suggest. Mutations in this gene also are the cause of some adult-onset ALS cases. However, FUS-associated ALS is more aggressive in children in whom these…