# NURTURE Study Design
Keywords: SMA, 5q SMA, Spinraza, CHOP INTEND, HINE-2, clinical trial, pre-symptomatic, motor milestones, respiratory intervention, SMN2 copies

> This page provides an overview of the NURTURE study, a Phase II clinical trial investigating the effects of Spinraza (nusinersen) in pre-symptomatic infants genetically diagnosed with 5q SMA, focusing on survival, motor milestones, and safety outcomes.

## Details
- [Patient Population](#): Pre-symptomatic infants genetically diagnosed with SMA, most likely to develop Type I or Type II, treated within 6 weeks of age.
- [Study Type](#): Open-label, Phase II, multinational, single-arm study.
- [Median Age at First Dose](#): Median age of participants at first dose was 22 days, ranging from 3 to 42 days.
- [Study Assessments](#): Primary endpoint focused on time to death or respiratory intervention, with additional endpoints including motor milestone scores and SMA progression.
- [Number of SMN2 Copies](#): Participants included 60% with 2 SMN2 copies and 40% with 3 SMN2 copies.
- [CHOP INTEND and HINE-2](#): CHOP INTEND evaluates motor skills in SMA Type I patients, while HINE-2 assesses developmental milestones in infants.
- [Post Hoc Analysis](#): A post hoc analysis excluded participants with specific baseline characteristics to align with criteria from other clinical trials on pre-symptomatic infants.
- [Adverse Event Reporting](#): Details on how to report adverse events in the UK and Ireland, including contact information for Biogen.