A single-dose gene replacement therapy is found to transform the capabilities for movement in children over 2 years of age and teenagers with spinal muscular atrophy, according to research published in Nature Medicine.

The effects allowed these minors who could sit but not stand to move like they’ve never done before, including standing up, walking, and even climbing stairs.

The real-world results of this phase 3 clinical trial, involving 126 children and adolescents, could support an alternative to lifelong, repeat-dose treatments for people who couldn’t get access to corrective treatment before 2, when curing the condition is possible.