In what experts are calling a “dream come true,” scientists used a recent biochemical discovery to help an 8-year-old boy with a rare genetic condition regain mobility.
Researchers from NYU Langone demonstrated, in a study published in Nature on Wednesday, how a chemical precursor to a commonly available enzyme, CoQ10, can help brain cells overcome a rare genetic condition that severely hobbles cells’ energy production process. Without treatment, the boy’s condition is known to deteriorate rapidly and could be fatal.
NYU Langone researchers have helped an 8-year-old boy regain mobility using an experimental treatment.