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Event-related brain potentials and working memory function in healthy humans after single-dose and prolonged intranasal administration of adrenocorticotropin 4-10 and desacetyl-alpha-melanocyte stimulating hormone

Cherqui S (2021) Hematopoietic stem cell gene therapy for cystinosis: from bench-to-bedside

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The study was designed as a pilot trial and included a relatively small number of participants, limiting its ability to detect differences in major outcomes such as death or hospitalization