Efzimfotase alfa, a second‑generation enzyme replacement therapy, was tested in a mouse model of hypophosphatasia (HPP) that has impaired mitochondrial respiration in skeletal muscles. Treatment significantly improved maximal respiration and spare respiratory capacity in muscle from affected mice, restoring these metrics close to wild‑type levels.
Efzimfotase alfa markedly improved skeletal muscle mitochondrial function in HPP mice, increasing maximal respiration and spare respiratory capacity toward wild‑type values and suggesting potential to improve muscular symptoms independent of skeletal manifestations
Akp2GW‑/‑ Knockout mouse (HPP model with loss of tissue‑nonspecific alkaline phosphatase)
Hypophosphatasia, mitochondrial dysfunction, enzyme replacement therapy, metabolic muscle impairment, respiratory capacity
Akp2GW‑/‑ constitutive Knockout of the Alpl gene, validation versus Akp2‑/‑ model, skeletal muscle respirometry assays, measurement of maximal respiration and spare respiratory capacity, Efzimfotase alfa treatment regimen
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