# CHERISH Study Design

Keywords: SMA, 5q SMA, CHERISH study, nusinersen, motor function, HFMSE, clinical trial, Type II SMA, Type III SMA, motor milestones

> This page provides detailed information about the CHERISH study, a Phase III clinical trial evaluating the efficacy of nusinersen in treating later-onset spinal muscular atrophy (SMA), focusing on motor function improvements and safety outcomes.

## Details
- [CHERISH Study Design](#): Overview of the Phase III trial involving 126 symptomatic patients with later-onset SMA, assessing nusinersen's impact on motor function and safety.
- [Natural History of Untreated SMA](#): Highlights the typical progression of untreated SMA, with patients experiencing a decline in motor function over time.
- [Clinically Meaningful Improvements](#): Explains the significant motor function improvements observed in patients treated with nusinersen compared to the sham-control group.
- [HFMSE](#): Describes the Hammersmith Functional Motor Scale—Expanded (HFMSE), a 33-item scale used to evaluate motor abilities in children.
- [Study Description](#): Detailed description of the CHERISH study methodology, including patient demographics, randomization, and primary and secondary endpoints.