Biogen has reported new interim results from NURTURE, an ongoing efficacy and safety study of Spinraza in 25 pre-symptomatic infants with spinal muscular atrophy (SMA).
The data was presented in a late-breaking session at the 23rd Annual Congress of the World Muscle Society (WMS) held in Mendoza in Argentina.
“The NURTURE study results demonstrate that early diagnosis and treatment with Spinraza has the potential to dramatically change the course of SMA,” said Wildon Farwell, Senior Medical Director, Clinical Development at Biogen.
“This is the longest available span of data on infants with SMA who began treatment in a pre-symptomatic period and indicates that children treated early with Spinraza can achieve motor milestones they would likely not attain without treatment.”
The interim analysis evaluated survival and respiratory intervention rates in infants who were genetically diagnosed with SMA and began treatment in the pre-symptomatic stage of the disease.
As of May 2018, all patients in the study were alive and none required tracheostomy or permanent ventilation.
Additionally, 22 of the 25 participants were able to walk either with assistance or independently according to the motor milestone standard of the World Health Organization and all 25 were able to sit without support.
Farwell added: “We continue to develop tools to inform our clinical research and are encouraged by the potential of neurofilament as a biomarker for SMA, how it could further expand the scientific understanding of this rare disease and, more importantly, its potential impact on those living with SMA.”