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Safety Profile from Clinical Trials The clinical trials reported that AOD-9604 displayed a very good safety and tolerability profile indistinguishable from placebo for most parameters
Systemic effects of inhaled corticosteroids
A 2026 retrospective longitudinal study using French national health claims data (20072020) identified 43 patients with LAL-D (17 infantile-onset, 26 later-onset), confirming that hepatomegaly, splenomegaly, and growth failure were the most frequent presenting features in infants and children, and highlighting the substantial healthcare utilization associated with both disease phenotypes (53)
Most current users inject AOD-9604 rather than taking it orally, so the trial data may underestimate what injectable protocols achieve
Most individuals can resume their daily activities within a few days
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