NAXD Deficiency: Heterogeneous Phenotypes and Positive Response to Niacin Treatment

Authors Seyedkatouli Najmesadat, Semcesen Liana N., Gallucci Lucia, Sikora Tim, Conrotte Jean‐François, Du Mei R. M., Kasakin Marat, Seferi Gezime, Corona Licia, Jakubec Martin, Nijagal Brunda, Lala Sajel, Ganetzky Rebecca D., Barreto Ana Maria Rodriguez, Szlago Marina, Wong Melanie, Shah Margit, Nurse James, Foulds Nicola, Sadagopan Shankar, Thu Ha Nguyen, Chi Dung Vu, Ngoc Khanh Nguyen, de Silva Michelle G., Ramialison Mirana, Rossello Fernando, Thorburn David R., Lynch Matthew, McGrath Pauline, Stroud David A., Christodoulou John, Linster Carole L., Van Bergen Nicole J.
Journal Journal of Inherited Metabolic Disease
Publication Date 2026-07-00
DOI 10.1002/jimd.70217

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