Sodium 4-phenylbutyrate reduces myofiber damage in a mouse model of Duchenne muscular dystrophy.

We performed a placebo-controlled pre-clinical study to determine if sodium 4-phenylbutyrate (4PB) can reduce contraction-induced myofiber damage in the mdx mouse model of Duchenne muscular dystrophy (DMD). At 72 h post-eccentric contractions, 4PB significantly increased contractile torque and reduced myofiber damage and macrophage infiltration. We conclude that 4PB, which is approved by Health Canada (Pheburane) and the United States Food and Drug Administration (Buphenyl) for urea cycle disorders, might modify disease severity in patients with DMD. [ABSTRACT FROM AUTHOR]

Dans cet article, on effectue une étude préclinique avec groupe placebo pour vérifier si le sodium 4-phénylbutyrate (« 4PB ») peut réduire les lésions myofibrillaires dues à la contraction chez la souris mdx, un modèle de la maladie de Duchenne de Boulogne (« DMD »). Soixante-douze heures après des contractions pliométriques, 4PB suscite une augmentation significative du moment de force contractile, diminue la lésion myofibrillaire et l'infiltration des macrophages. En conclusion, 4PB, approuvé par Santé Canada (Pheburane) et FDA (Buphenyl) pour le traitement des troubles du cycle de l'urée, pourrait modifier la gravité de la maladie chez les patients aux prises avec la DMD. [Traduit par la Rédaction] [ABSTRACT FROM AUTHOR]

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