Duchenne
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Related Articles from SNS
Dad’s warning after son, 12, finally gets 'life-changing' Duchenne drug
Dad’s warning after son, 12, finally gets 'life-changing' Duchenne drug The drug givinostat - used for Duchenne Muscular Dystrophy management - remains unavailable to boys and young men who can no longer walk and this had plunged Alex Clarke's family into uncertainty A 12-year-old boy with a rare muscle-wasting condition has finally started a life-changing NHS treatment after years of uncertainty for his family. Ben Clarke has Duchenne Muscular Dystrophy (DMD), a severe, progressive genetic...
Pathology-Targeted EP4 Agonism Reverses Fibrosis in a Rat Model of DMD
Duchenne muscular dystrophy (DMD) presents a critical therapeutic gap in adolescent patients, where fibro-fatty muscle replacement and depletion of the regenerative niche render existing interventions insufficient. Prostaglandin E2 signaling through the EP4 receptor stimulates muscle regeneration, but systemic off-target effects have limited clinical translation of EP4 agonism. We evaluated irodanoprost (IROD), a bone-targeted prodrug of an EP4-selective agonist, in a DMD rat model using...