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AAV-mediated genetic interventions in the treatment of rare kidney disease: a narrative review
Ago 24, 2026
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Orphanet Journal of Rare Diseases
Orphanet Journal of Rare Diseases
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Correction: Caregiver-reported disease burden in Krabbe disease: evaluating outcomes of hematopoietic stem cell transplantation
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Invisible impairments? A study of emotional and social functioning in children with inborn errors of metabolism
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