Two Medical Breakthroughs for Spinal Muscular Atrophy Treatment
Treatment with gene therapy
For the gene therapy method, the treatment involves copies of the SMN1 gene that are working together with viruses like AAV9. It is then introduced in an infusion that is given intravenously. The 3 children who were given a low dose of this medication showed a slight improvement. However, the 12 children who received higher dosages were able learn new activities, such as sitting upright, rolling over, eating, speaking, and walking a few steps, that they weren't able to do before. These skills are considered big feats for those who have SMA.