A two-year, real-world outcome analysis reported durable gains in infants with severe SMA following early gene therapy, reinforcing the translational signal that timing and dosing can preserve function long beyond trial endpoints. The evidence comes from the Italian national registry and is designed to reflect routine-care variation. In a separate rare neurobiology and translational platform update, a bone marrow-on-a-chip model with NIH support enabled ex vivo study of plasma cell development and migration across staged subniches. Published in Science Advances, the platform aims to better reproduce the microenvironments governing antibody-secreting cell fate, supporting immunotherapy and rare immunodeficiency research. The combined picture is a push toward both durable in vivo correction for neurogenetic disease and better ex vivo systems to interrogate immune cell behavior driving therapeutic response.