Reduction in ABR between the treatment arms was observed regardless of baseline subgroups examined, including age, presence or absence of target joints, and pre-trial treatment regimen
Bondue T, Berlingerio SP, Siegerist F, Sendino-Garvi E, Schindler M, Baelde HJ, Cairoli S, Goffredo BM, Arcolino FO, Dieker J, Janssen MJ, Endlich N, Brock R, Gijsbers R, van den Heuvel L, Levtchenko E (2023) Evaluation of the efficacy of cystinosin supplementation through CTNS mRNA delivery in experimental models for cystinosis
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179 Lactate accumulation under hypoxia further amplifies this cascade, reinforcing metabolic adaptation