Two Medical Breakthroughs for Spinal Muscular Atrophy Treatment
What were the results of this treatment?
After a year and 4 months of giving the treatment, the 15 children were still living. This is high as compared to the 8% survival rate for patients who did not have any treatment. And moreover, the children who survived did well continuously for two years.
The children who were the healthiest took tentative steps, went to a doctor to be diagnosed before symptoms appeared, and had brothers and sisters who had SMA. The result of this study has shown importance in the continuous improvement of newborn screening. There is even a case of a young girl that went through a successful SMA gene therapy.
