An infant with spinal muscular atrophy type 1 became the first baby to receive an experimental drug, nusinersen, to treat her condition in 2013, according to Medical Xpress.
The patient received the treatment as part of a phase-2 clinical trial, which involved 20 children. On Dec. 6, 2016, The Lancet published the phase-2 trial results. Cambridge, Mass.-based Biogen and Carlsbad, Calif.-based Ionis Pharmaceuticals developed nusinersen.
Here are five things to know:
1. Pediatric neurologist Richard Finkel, MD, of Orlando, Fla.-based Nemours Children's Hospital, led the study, with pediatric neurologist John Day, MD, PhD, director of the Neuromuscular Disorders Clinic at Lucile Packard Children's Hospital Stanford in Palo Alto, Calif., as well as a neurology and pediatrics professor at the Stanford University School of Medicine, as a co-author.
2. The study found nusinersen to be a safe and well-tolerated drug.
3. The researchers also discovered patients receiving the drug experienced improvements in motor and nerve function.
4. A phase-3 trial testing nusinersen for spinal muscular atrophy type 1 patients found "infants receiving the drug were achieving significantly more motor milestones than those in the control group," according to Medical Xpress. Therefore, the trial was halted in August 2015, and is now considered open-label, where all participants receive nusinersen as opposed to testing against a control group.
5. FDA approval for nusinersen is anticipated in the next two months.