Outcomes after 1-year in presymptomatic infants with genetically diagnosed spinal muscular atrophy (SMA) treated with nusinersen: interim results from the NURTURE study

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NURTURE (NCT02386553) is an ongoing phase 2, open-label, single-arm study, evaluating intrathecal nusinersen (12-mg equivalent dose) in infants with presymptomatic SMA. Enrolled infants were presymptomatic at screening, age ≤6 wks at first dose, had genetic diagnosis of SMA and 2 or 3 SMN2 copies. The primary endpoint is time to death or respiratory intervention (tracheostomy or ventilation for ≥6 hours/day for ≥7 days). Secondary endpoints include: proportion of infants developing protocol-defined SMA symptoms (defined by age-adjusted weight, g-tube placement or failure to achieve appropriate WHO motor milestones), motor milestone attainment assessed by Hammersmith Infant Neurological Examination (HINE) Section 2 and change in Children’s Hospital of Philadelphia Infant Test of Neuromuscular Disorders (CHOP INTEND) scale.

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