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Identifying novel response markers for spinal muscular atrophy revealed by targeted proteomics following gene therapy.
Gene Ther
January 2025
Departments of Pediatrics and Neurology, Emory University, Atlanta, 30322, Georgia.
Spinal muscular atrophy (SMA) is a progressive disease that affects motor neurons, with symptoms usually starting in infancy or early childhood. Recent breakthroughs in treatments targeting SMA have improved both lifespan and quality of life for infants and children with the disease. Given the impact of these treatments, it is essential to develop methods for managing treatment-induced changes in disease characteristics.
View Article and Find Full Text PDFMotor function and compound muscle action potential amplitude in children with spinal muscular atrophy treated with nusinersen.
Brain Dev
January 2025
Department of Neurology Children's Hospital of Chongqing Medical University, National Clinical Research Center for Child Health and Disorders, Ministry of Education Key Laboratory of Child Development and Disorders, Chongqing Key Laboratory of Child Neurodevelopment and Cognitive Disorders, Chongqing, China. Electronic address:
Background: Disease-modifying therapies can improve motor function in patients with spinal muscular atrophy (SMA), but efficacy varies between individuals. The aim was to evaluate the efficacy and safety of nusinersen treatment in children with SMA and to investigate prognostic factors.
Methods: Motor function, compound muscle action potential (CMAP), and other indicators were prospectively collected before and 14 months after nusinersen treatment.
Predicting purulent meningitis in very preterm infants: a novel clinical model.
BMC Pediatr
January 2025
Department of Pediatrics, Qilu Hospital, Shandong University, No.107, West Culture Road, Lixia District, Jinan City, Shandong Province, 250000, China.
Background: Purulent meningitis (PM) is a commonly encountered infectious condition in newborns, which unfortunately can result in infant mortality. Newborns with PM often present nonspecific symptoms. The success of lumbar puncture, an invasive test, relies on the operator's expertise.
View Article and Find Full Text PDFAltered 3D genome reorganization mediates precocious myeloid differentiation of aged hematopoietic stem cells in inflammation.
Sci China Life Sci
December 2024
Clinical and Translational Research Center of Shanghai First Maternity & Infant Hospital, Frontier Science Center for Stem Cells, School of Life Sciences and Technology, Tongji University, Shanghai, 200092, China.
Inflammation is a driving force of hematopoietic stem cells (HSCs) aging, causing irreversible exhaustion of functional HSCs. However, the underlying mechanism of HSCs erosion by inflammatory insult remains poorly understood. Here, we find that transient LPS exposure primes aged HSCs to undergo accelerated differentiation at the expense of self-renewal, leading to depletion of HSCs.
View Article and Find Full Text PDFDetailed analysis of cystic lesions in patients after open fetal repair and postnatal myelomeningocele closure.
Childs Nerv Syst
January 2025
Division of Neurosurgery, Department of Surgery, Children's Hospital of Philadelphia, Philadelphia, PA, USA.
Purpose: We sought to evaluate the incidence, natural history, and management of cystic spinal lesions following myelomeningocele/myeloschisis closure.
Methods: We performed a single-center retrospective review of all patients who underwent myelomeningocele/myeloschisis closure from 2013 to 2018 with follow-up to 5 years old.
Results: We analyzed 100 fetal repairs and 81 postnatal closures from 305 total surgeries.
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