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Neuromodulators (Botox, Dysport, Nuceiva) begin onset between day three and seven, peak at day fourteen, and hold for 12 to 16 weeks for most patients
Similar to other lcFAODs, hepatic symptoms are only seen during (early) childhood and patients with onset of symptoms at a later age predominantly experience myopathy-related complaints
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The compound was originally developed by Ipsen but was licensed to Rhythm Pharmaceuticals in 2010 (67, 68)
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