9-Jul-2025
Experimental compound offers potential treatment for rare, often fatal childhood disease
NYU Langone Health / NYU Grossman School of MedicinePeer-Reviewed Publication
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.
- Journal
- Nature
- Funder
- NIH/National Institutes of Health, NIH/National Institutes of Health, Perlmutter Cancer Center, Damon Runyon Cancer Research Foundation, Dale Frey Breakthrough Award, Tara Miller Melanoma Foundation, American Cancer Society, Harry J. Lloyd Charitable Trust, Irma T. Hirschl Trust, Concern Foundation