, 2019). ERT relies on periodic infusions, normally requiring the use of intra‐venous units that are linked to chance of lifestyle‐threatening infections. Consequently, the effect on the quality of life of people as well as their caregivers is sizeable (Wyatt et al (2015) A murine Niemann‐Decide on C1 I1061T knock‐in https://hankd432thr6.glifeblog.com/profile