Today Biogen provided us with an update on the NURTURE study which was published in Muscle & Nerve¹.

New results continue to show that children who started the treatment before onset of symptoms continue to maintain and achieve new motor milestones. In addition the treatment continue to show good safety profile.

The study does not show any death or need for permanent ventilation in the children. And 23 out 25 are now walking independently. Overall, 22 of 25 children in the study (12 with two copies of the SMN2 gene and 10 with three copies of the SMN2 gene) achieved the maximum score on the Children’s Hospital of Philadelphia Infant Test of Neuromuscular Disorders.

The NURTURE data also provided further key insights on the growing importance of objective measures of disease activity and the evolving definition of pre-symptomatic SMA.

Additional details can be found here:

Continued benefit of nusinersen initiated in the presymptomatic stage of spinal muscular atrophy: 5‐year update of the NURTURE study – Crawford – Muscle & Nerve – Wiley Online Library

About the trial

NURTURE is an ongoing, Phase 2, open-label study of 25 pre-symptomatic patients with the genetic diagnosis of SMA (considered most likely to develop SMA Type 1 or 2) who received their first dose of nusinersen before six weeks old. The study is evaluating the longer-term efficacy and safety ofnusinersen through eight years of age to further understand the impact of early treatment. More information on the NURTURE study (NCT02386553) is available on clinicaltrials.gov.