SMA: Wider ‘Window of Opportunity’ for Treatment?
Quest | August 4, 2011 | By Margaret Wahl
The “window of opportunity” for treating infants at risk of developing spinal muscular atrophy (SMA) may not be as narrow as some experts have feared, new experiments in mice suggest.
MDA research grantee Umrao Monani at Columbia University Medical Center in New York coordinated the scientific team, which published its results Aug. 1, 2011, in the Journal of Clinical Investigation. (See Postsymptomatic restoration of SMN rescues the disease phenotype in a mouse model of severe spinal muscular atrophy.)
Monani described the “window of opportunity” as “the period between the birth of the child and the last point in time during which the motor unit [nerve cell and the muscle fibers it activates] can be rescued.”
This window may be “open” for a few weeks or a few months after birth, and may extend into the period when the child begins to show SMA symptoms, Monani said. “I suspect that if the patient can be treated during this period, he or she will benefit greatly.”
This finding, if confirmed in humans, further bolsters the case for newborn screening of infants for SMA. It also is good news for the potential use of several treatments for SMA currently in development.
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