In a study supported by the National Institute of Health (NIH), researchers at Iowa State University found that a drug called A15/283 helped to ameliorate symptoms of a mild form of spinal muscular atrophy (SMA) in mice.
SMA is a rare, degenerative, genetic disorder that results in weakened and atrophied muscles. To learn more about SMA click here.
Professor Ravindra Singh’s lab treated mice on the first and third days after they were born. Results showed a significant improvement in the mice’s symptoms. These results also shed light on how male and females react differently to SMA treatment. Singh has aspirations for this drug to move to human clinical trials, but recognizes more research needs to be done before this happens.
While there is obviously more work to complete, this professor’s hope for the potential SMA treatment means hope for the rare disease community.
To learn more about the study in Newswise, click here and in the US National Library of Medicine National Institutes of Health, click here.
