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The approval is supported by positive results from the largest and longest phase 3 study for gene therapy in hemophilia, showing a 52% reduction in annualized bleeding rate compared with baseline rates on routine prophylaxis.
Significant improvements seen in hematologic parameters, visceral volumes, and plasma biomarkers in patients with Gaucher disease, suggesting its potential as an alternative therapy, particularly for those who cannot access enzyme replacement therapy.
New data quantify the exposure to wildfire particulate matter among schoolchildren in California, finding that interventions in a small number of schools may reduce the overall burden of wildfire PM2.5 exposure.
A silent disease characterized as a chronic cutaneous neuropathy, notalgia paresthetica has an unmet need yet to be fully understood and, dermatologists argue, an FDA-approved treatment could help improve understanding of the etiology and prevalence of this disease.
Built environments, but not social and economic environments, were negatively associated with BMI and overweight or obesity status, with a stronger association among adolescents with longer exposure to their built environment.