New registry analysis suggests gene therapy can deliver durable, two-year gains in infants with severe spinal muscular atrophy, adding to evidence that early-life delivery may translate into sustained function. The findings, published in Annals of Clinical and Translational Neurology, reflect real-world outcomes rather than solely trial populations. Meanwhile, an international consensus effort in Annals of Hematology established detailed guidance for safely delivering CPX-351 outside the hospital ward for patients with therapy-related AML and AML-MRC. For clinicians, the document supports operational scaling of a liposomal cytarabine/daunorubicin approach in more settings. Together, the items show both biological durability work in neurogenetics and pragmatic care-delivery expansion in hematology.