Impressive! The results of NURTURE, a presymptomatic clinical trial of Spinraza, are out. The drug appears to prevent SMA altogether in the majority of children when started early!
On the 2nd of October Biogen released data from the NURTURE study of infants with spinal muscular atrophy treated pre-symptomatically with Spinraza.
The trial has been running since 2015. Twenty-five asymptomatic newborns with genetically confirmed SMA (and 2 or 3 copies of the SMN2 gene) were quickly put on Spinraza and monitored regularly. These results are after at least 24 months observation.
All of the children are alive and well with none requiring further ventilation. All are also able to sit unsupported and twenty-two of them are walking!
Please click here to read detailed results.
TreatSMA is working hard to bring about routine newborn screening for SMA in the UK, so that the disease is detected and treated before it gives symptoms.
