When 2020 began, Neena Nizar and her family were poised to harvest the fruit of a decade of hard work and sacrifice: a clinical trial of an experimental treatment for her two sons’ rare genetic disorder that was slated to start before the year’s end.
“I can’t even put into words what we’ve been able to do to get to this point,” she says. “My kids have given bone biopsies; I gave up my job and moved to a new country. We’ve just been going, going, going.”
