New data from the phase 2 NURTURE trial evaluating nusinersen (Spinraza®; Biogen) demonstrate that presymptomatic patients with spinal muscular atrophy (SMA) continue to benefit from treatment for up to 4.8 years. 

NURTURE is an ongoing, open-label, multinational study assessing 25 infants with the genetic diagnosis of SMA (most likely to develop SMA Type 1 and 2) who received their first dose of nusinersen in the presymptomatic stage and before 6 weeks old. The primary end point of the study is time to death or respiratory intervention, defined as invasive or noninvasive ventilation for ≥6 hours/day continuously for ≥7 days or tracheostomy.

Interim study analysis as of February 2020 showed that all patients (median age of 3.8 years) were alive without the need for permanent ventilation. Additionally, nusinersen demonstrated sustained benefit over the 11 months of follow-up since the last data cut. 

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All patients who were previously able to walk with assistance (92%) and walk independently (88%) maintained that ability through the last visit. One patient also gained the ability to walk with assistance and achieved a maximum score on the CHOP-INTEND (Children’s Hospital of Pennsylvania Infant Test of Neuromuscular Disorders) scale over the 11 months. At data cutoff, 21 patients (84%) achieved a maximum score on the CHOP-INTEND scale.

With regard to safety, nusinersen was well-tolerated with no new safety concerns. Findings from the study will be presented at the virtual Cure SMA Research & Clinical Care Meeting on June 10-12, 2020.

“The impact of early and sustained Spinraza treatment on these infants and their families is remarkable,” said Kathryn Swoboda, MD, the Katherine B. Sims, MD, Endowed Chair in Neurogenetics and Director of the Neurogenetics Program, Massachusetts General Hospital. “The new results from NURTURE continue to bolster the substantial benefit of both prompt diagnosis and early and longer-term treatment with Spinraza.”

Biogen also announced that the NURTURE study has been extended an additional 3 years in order to evaluate the long-term safety and efficacy of the drug through 8 years of age.

Spinraza, a survival motor neuron-2 (SMN2)-directed antisense oligonucleotide, is currently approved for the treatment of SMA in pediatric and adult patients. 

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This article originally appeared on MPR