Columbia-based University of Missouri researchers are studying spinal muscular atrophy with respiratory distress type 1, according to Health Canal.
The researchers created a gene replacement therapy to treat and control SMARD1. They tested the therapy, which can cross the blood-brain barrier, on mice with SMARD1.
Here are six insights:
1. A low dose of the gene-replacement therapy resulted in muscle strength improvements and motor neuron protein expression.
2. The therapy also extended mice life span.
3. Researchers conclude the gene-replacement therapy may potentially treat SMARD1.
4. Next steps involve identifying the exact dose needed. Researchers also want to pinpoint when they should administer the therapy in the disease cycle and where in the body they should administer the therapy for the greatest impact.
5. SMARD1 currently does not have a cure and the average life expectancy of children with the disease is 13 months.
6. SMA impacts about 200,000 Americans.