Gene Therapy Now Leads Spinal Muscular Atrophy Treatment as Real-World Sequencing Patterns Emerge
A large US claims analysis shows that after widespread newborn screening, gene replacement therapy has become the dominant first treatment ...
Tag: spinal muscular atrophy
Sleep Lab Thresholds Under Scrutiny: When Should Children With Muscle Disease Start Ventilation?
A new letter in the Journal of Clinical Sleep Medicine scrutinizes the polysomnographic thresholds used to decide when children with ...
Stress-Triggered SMN Droplets Act as a Rescue System for Misplaced Splicing Machinery
Researchers in Bonn have discovered that the SMN protein forms reversible cytoplasmic condensates under stress to trap and clear misplaced ...
Approved Metabolism Drugs Rescue Disease Signs in SMA Mice
Three commercially approved metabolism-targeting drugs—pioglitazone, melatonin and insulin—improved survival, motor function, muscle size and hepatic pathology in Smn2B/- spinal muscular ...
Gene Therapy Shows Lasting Two-Year Gains in Infants With Severe Spinal Muscular Atrophy
A two-year follow-up of 64 Italian children shows that gene therapy with onasemnogene abeparvovec continues to deliver motor gains well ...
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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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