AANS Neurosurgeon | Volume 26, Number 4, 2017

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Drug Improves Muscle Function and Survival in Spinal Muscular Atrophy

Final results from a phase 3 clinical trial of nusinersen (Spinraza) published in NEJM

More than half of the babies with infantile-onset spinal muscular atrophy (SMA) who were treated with nusinersen (Spinraza) gained motor milestones, compared to none of the babies in the control group. Infants treated with the drug also had 63 percent lower risk of death. 

Babies born with SMA, a genetic disorder affecting nerves that control muscle movement, cannot hold up their heads, roll over or sit up independently. They often do not survive beyond 2 years of age.

“This landmark study provides the most robust type of evidence that nusinersen, which targets the genetic defect in SMA, can safely and effectively help infants with this devastating condition gain muscle function,” said study co-author Nancy Kuntz, MD, Principal Investigator at Ann & Robert H. Lurie Children’s Hospital of Chicago, Medical Director of Mazza Foundation Neuromuscular Program and Professor of Pediatrics and Neurology at Northwestern University Feinberg School of Medicine.

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