About Super Baby

Spinal muscular atrophy (SMA) is a rare, inherited disease that results in loss of nerves in the spinal cord and weakness of the muscles connected with those nerves. The muscles most frequently affected are those of the neck and trunk that control posture, those of the legs and arms that control movement, and those in the area of the ribs that help breathing. People with SMA generally appear normal at birth; the symptoms develop as early as 3 months in the most severely affected, around 1 to 2 years of age in the moderately affected, and more rarely in late childhood or adult years in mildly affected individuals.

There is no known treatment for SMA; historically, nearly half of babies born with the most severe form of the disease have died before two years of age.  All people with SMA have a higher than normal risk for progressive disability.  The most severely affected are at risk for breathing complications and early death.

NN101, a biomarker-driven study, was the first funded NeuroNEXT project. The goal was to identify laboratory measures to improve SMA clinical trials. Enrollment began in November, 2012 and ended September, 2014. Enrollment of all healthy control infants was completed in less than one year; enrollment of infants with SMA also occurred ahead of schedule. Retention was excellent and we have learned essential clinical trial lessons from the Super Babies!