Youngest UK ALS trial participant

A 14-year-old boy, Kyle Sieniawski, is the youngest UK resident with amyotrophic lateral sclerosis (ALS) joining a rare trial linked to the fused in sarcoma (FUS) gene at King’s College Hospital. The trial tests ulefnersen (formerly Ion363) to slow or halt progression in people with mutations in the FUS gene. Participants receive the drug or a placebo every three months by lumbar puncture, in the hope it helps children and others with this ALS.