Orlando, Fla.-based Nemours Children's Hospital researchers studied the impact of a new medication nusinersen on infants with severe spinal muscular atrophy.
The study involved 121 infants with SMA Type 1. The infants either received nusinersen injections or a sham treatment.
New England Journal of Medicine published the study.
Here are eight takeaways:
1. Nusinersen's effects were so impressive during the study the FDA approved the medication earlier than anticipated, in 2016. The drug is available as Spinraza.
2. With an antisense oligonucleotide, nusinersen modifies the SMN2 gene, which is injected into the spinal fluid. The spinal cord's nerve cells then absorb the DNA to boost neuromotor development protein production.
3. Over 13 months, 41 percent of the nusinersen group saw improvements in motor function. Some of the infants started developing the following skills: kicking, head control, rolling over and standing.
4. No infants in the control group saw motor skill improvement.
5. The death risk proved 63 percent lower for the nusinersen group compared to the control group.
6. The nusinersen-treated infants were 47 percent more likely to survive without permanent assisted ventilation.
7. The infants treated with nusinersen mostly tolerated the drug, and researchers noted no serious safety issues associated with the drug.
8. Other studies, testing aspects such as the long-term effects of the drug, are now occurring.