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Biomarkers to predict disease progression and therapeutic response in isolated methylmalonic acidemia.
Manoli, Irini; Gebremariam, Abigael; McCoy, Samantha; Pass, Alexandra R; Gagné, Jack; Hall, Camryn; Ferry, Susan; Van Ryzin, Carol; Sloan, Jennifer L; Sacchetti, Elisa; Catesini, Giulio; Rizzo, Cristiano; Martinelli, Diego; Spada, Marco; Dionisi-Vici, Carlo; Venditti, Charles P.
Affiliation
  • Manoli I; Metabolic Medicine Branch, National Human Genome Research Institute, National Institutes of Health (NIH), Bethesda, Maryland, USA.
  • Gebremariam A; Metabolic Medicine Branch, National Human Genome Research Institute, National Institutes of Health (NIH), Bethesda, Maryland, USA.
  • McCoy S; Metabolic Medicine Branch, National Human Genome Research Institute, National Institutes of Health (NIH), Bethesda, Maryland, USA.
  • Pass AR; Metabolic Medicine Branch, National Human Genome Research Institute, National Institutes of Health (NIH), Bethesda, Maryland, USA.
  • Gagné J; Metabolic Medicine Branch, National Human Genome Research Institute, National Institutes of Health (NIH), Bethesda, Maryland, USA.
  • Hall C; Metabolic Medicine Branch, National Human Genome Research Institute, National Institutes of Health (NIH), Bethesda, Maryland, USA.
  • Ferry S; Metabolic Medicine Branch, National Human Genome Research Institute, National Institutes of Health (NIH), Bethesda, Maryland, USA.
  • Van Ryzin C; Metabolic Medicine Branch, National Human Genome Research Institute, National Institutes of Health (NIH), Bethesda, Maryland, USA.
  • Sloan JL; Metabolic Medicine Branch, National Human Genome Research Institute, National Institutes of Health (NIH), Bethesda, Maryland, USA.
  • Sacchetti E; Division of Metabolic Diseases, Bambino Gesù Children's Hospital IRCCS, Rome, Italy.
  • Catesini G; Division of Metabolic Diseases, Bambino Gesù Children's Hospital IRCCS, Rome, Italy.
  • Rizzo C; Division of Metabolic Diseases, Bambino Gesù Children's Hospital IRCCS, Rome, Italy.
  • Martinelli D; Division of Metabolic Diseases, Bambino Gesù Children's Hospital IRCCS, Rome, Italy.
  • Spada M; Division of Hepato-Bilio-Pancreatic Surgery, Liver and Kidney Tranplantation, Bambino Gesù Children's Hospital, IRCCS, Rome, Italy.
  • Dionisi-Vici C; European Research Network TransplantChild, Rome, Italy.
  • Venditti CP; Division of Metabolic Diseases, Bambino Gesù Children's Hospital IRCCS, Rome, Italy.
J Inherit Metab Dis ; 46(4): 554-572, 2023 07.
Article in En | MEDLINE | ID: mdl-37243446

Full text: 1 Collection: 01-internacional Database: MEDLINE Main subject: Amino Acid Metabolism, Inborn Errors Type of study: Diagnostic_studies / Guideline / Prognostic_studies / Risk_factors_studies Limits: Humans Language: En Journal: J Inherit Metab Dis Year: 2023 Document type: Article Affiliation country: United States Country of publication: United States

Full text: 1 Collection: 01-internacional Database: MEDLINE Main subject: Amino Acid Metabolism, Inborn Errors Type of study: Diagnostic_studies / Guideline / Prognostic_studies / Risk_factors_studies Limits: Humans Language: En Journal: J Inherit Metab Dis Year: 2023 Document type: Article Affiliation country: United States Country of publication: United States