Babies identified with SMA within the first one to two weeks of life can receive a one-time gene therapy infusion leading to significantly improved life outcomes.

“so we started out a newborn screening program for SMA and then babies that were identified through that had the option if they wanted to of going into a clinical trial that ended up being quite synergistic in the sense that we did identify babies who would have been predicted to have the most severe type of SMA they did get into early clinical trials right away they did benefit from those that helped in terms of the ultimate evidence that was necessary to show the efficacy of those treatments and because of that um and because we were able to show that we could do it technically and that people wanted it um SMA has been added to the recommended Universal screening panel for babies across the United States and so now 4 million babies born each year in the United States are screened for SMA and we have three FDA approved treatments including a oneandone gene therapy so babies can now be identified within the first week or two of Life get a oneandone IV infusion of the genene and uh go on to have a a much much better life if not uh you know be quote unquote normal at least as far as we can see so far”