Page 18 - Mobility Management, February 2018
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Cure SMA: Children on Nusinersen More Likely to Hit Milestones
A new study published in the New England Journal of Medicine found that infants who have Type 1 spinal muscular atrophy (SMA) and took the drug nusinersen (brand name: Spinraza) were far more likely to hit motor milestones than infants with SMA who were not given the treatment.
Researchers used the Hammersmith Infant Neurological Examination to determine improvements in motor milestone categories, and they also tracked when study participants needed permanent ventilation and/or when they died.
The study showed that 51 percent of infants on Spinraza “were motor milestone responders, compared to untreated infants,” none of whom achieved motor milestones, according to a news announcement from Cure SMA.
There was also a “statistically signi cant 47 percent
reduction in the risk of death or use of permanent assisted ventilation,” the announcement said.
Cure SMA said Spinraza “demonstrated a favorable bene t-risk pro le.”
In December 2016, nusinersen became the  rst drug therapy FDA approved to treat SMA, which is the world’s leading genetic cause of death in infants.
Cure SMA’s annual conference, for SMA families and researchers, is June 14-17 in Dallas. m
Study: Heart
Function Is
Impacted by
Spinal Cord Injury
Researchers at the University of British Columbia have determined that spinal cord injuries (SCI) affect the heart.
In research published in Experimental Physiology, Christopher West, Ph.D., assistant professor of kinesi- ology at the University of British Columbia, compared two severities of SCI in rodents. West and the research team concluded that SCI causes changes in heart function. The extent of those changes depends on how severe the SCI is; a relatively small number of healthy nerve  bers is necessary for the heart to function at a near-normal level.
The research team said its heart study “has important clinical implications as one of the primary aims of spinal cord injury management is to reduce the amount of damage that happens after the initial trauma. This exac- erbation of the primary injury is termed the secondary injury, and is driven by complex cellular processes that eventually result in the ‘size’ of the injury enlarging and therefore damaging more nerve  bers.”
The study suggests that a patient’s heart function could be improved if secondary injury can be reduced, which would lead to the preservation of more nerve  bers. m
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