Drug Trial Gives Hope to Children with SMA

At the Cure SMA Annual Spinal Muscular Atrophy Conference held at Disneyland in Anaheim, CA this past weekend, new results from the NURTURE study were reported by Dr. Darryl C. De Vivo, adding data to the longest study of spinal muscular atrophy (SMA) in pre-symptomatic infants (n=25).  According to a release issued by Biogen, these data continue to demonstrate efficacy and safety in patients treated pre-symptomatically with SPINRAZA in comparison to the natural history of this disease.  “We are seeing an extensive number of patients continually meeting child motor development milestones and making unprecedented gains in a previously hopeless and often fatal condition,” said Dr. De Vivo, adding that the treatment has now been shown to “improve outcomes for patients of all ages.”  The story of an SMA family treated by Dr. De Vivo was released nationwide in a CBS Newspath segment, highlighting the study’s remarkable results. 

Also covered by: WCAX and MedCity News.