The value of a recent biochemical discovery can be seen in the case of an 8-year-old boy, who was playing typical sports in August 2023, but by November needed a wheelchair because of a rare disease that caused worsening paralysis. As part of a new study, neurologists at NYU Langone Health treated the boy with an experimental compound that partially reversed his rapid decline. Two months after beginning treatment, he was able to walk long distances again and even run.