FAERS analysis identifies platelet and triglyceride signals for givinostat
A retrospective FAERS analysis of 321 givinostat reports identified platelet abnormalities and increased blood triglycerides as prominent safety signals but ...
Tag: Duchenne muscular dystrophy
Musculoskeletal shear-wave elastography in the paediatric population: a systematic review
A systematic review of 67 studies involving 2,558 children found that shear-wave elastography shows promise for assessing and monitoring musculoskeletal ...
Spanish survey finds high DMD gene-therapy willingness amid readiness gaps
A cross-sectional survey of 53 Spanish healthcare professionals found high willingness to adopt Duchenne muscular dystrophy gene therapy, alongside concerns ...
Non-invasive adjunctive diagnostic models incorporating high-frequency ultrasound and shear wave elastography for pediatric Duchenne muscular dystrophy
A retrospective study of 114 children found that a model combining high-frequency ultrasound and shear-wave elastography had higher diagnostic discrimination ...
Mixed-methods study finds equivalent remote NSAA scores in 18 people with Duchenne muscular dystrophy
A mixed-methods study of 18 people with Duchenne muscular dystrophy reported equivalent NSAA scores across live telehealth, asynchronous at-home video ...
Brain imaging in dystrophinopathies
A comprehensive review in the Journal of Neurology synthesizes decades of brain imaging evidence showing that Duchenne and Becker muscular ...
Machine Learning Links a Rare Protein Modification and Mast Cells to Duchenne Muscular Dystrophy
A new multi-omics and machine learning study identifies the USP18 gene as a hub linking isonicotinylation and mast cell activity ...
Micro-Dystrophin Gene Therapy Reveals Hidden Immune Storm in Treated Boys
A proteomic study of the phase 1/2a delandistrogene moxeparvovec trial shows that high-dose AAV gene therapy triggers a sharp but ...
From First Breath to Lifelong Care: How Newborn Screening Is Being Rebuilt Around Every Child
The Society for Pediatric Research's 2026 Mary Ellen Avery Award recognizes Wendy Chung's work building population-scale newborn screening systems, from ...
A Father’s Two-Decade Journey Through Duchenne Muscular Dystrophy Reshapes What Research Means
A new Pediatric Research reflection by Alex Smith traces nearly twenty years of living alongside his son Harrison's Duchenne muscular ...
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Mothers who receive childcare support from maternal grandparents show more parental warmth, finds NTU Singapore study
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University of Seville Breaks 120-Year-Old Mystery, Revises a Key Einstein Concept
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Bee body mass, pathogens and local climate influence heat tolerance
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Researchers record first-ever images and data of a shark experiencing a boat strike
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Groundbreaking Clinical Trial Reveals Lubiprostone Enhances Kidney Function
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