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Oct 20: Nearly two years ago, the FDA approved BioMarins Voxzogo (vosoritide) as the first therapy for achondroplasia, a rare genetic disorder that causes the most common form of dwarfismfueling ongoing controversy within the dwarfism community about such treatments

ARA290 works by selectively activating specific receptors that trigger protective cellular pathways without affecting hematocrit levels

Updated June 20, 2025

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If methemoglobinemia is life-threatening, methylene blue administration has still been suggested to be the first-line treatment in G6PD-deficient subjects, starting at a lower dose of 0.3 to 0.5 mg/kg and titrating upward to further reduce methemoglobinemia [14, 20]