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Biochemical and clinical response after umbilical cord blood transplant in a boy with early childhood-onset beta-mannosidosis.
Lund, Troy C; Miller, Weston P; Eisengart, Julie B; Simmons, Katrina; Pollard, Laura; Renaud, Deborah L; Wenger, David A; Patterson, Marc C; Orchard, Paul J.
Afiliación
  • Lund TC; Division of Pediatric Blood and Marrow Transplant, University of Minnesota, Minneapolis, Minnesota.
  • Miller WP; Sangamo Therapeutics, Richmond, California.
  • Eisengart JB; Division of Clinical Behavioral Neuroscience, Department of Pediatrics, University of Minnesota, Minneapolis, Minnesota.
  • Simmons K; Sanofi, Rare Disease Division, Sanofi Genzyme US, Bridgewater, New Jersey.
  • Pollard L; Biochemical Genetics Laboratory, Greenwood Genetic Center, Greenwood, South Carolina.
  • Renaud DL; Department of Neurology, Department of Clinical Genomics, Department of Pediatrics, Mayo Clinic, Rochester, Minnesota.
  • Wenger DA; Lysosomal Diseases Testing Laboratory, Department of Neurology, Sidney Kimmel Medical College, Thomas Jefferson University, Philadelphia, Pennsylvania.
  • Patterson MC; Division of Child and Adolescent Neurology, Mayo Clinic, Rochester, Minnesota.
  • Orchard PJ; Division of Pediatric Blood and Marrow Transplant, University of Minnesota, Minneapolis, Minnesota.
Mol Genet Genomic Med ; 7(7): e00712, 2019 07.
Article en En | MEDLINE | ID: mdl-31115173

Texto completo: 1 Base de datos: MEDLINE Asunto principal: Trasplante de Células Madre de Sangre del Cordón Umbilical / Beta-Manosidosis / Beta-Manosidasa Tipo de estudio: Diagnostic_studies Idioma: En Revista: Mol Genet Genomic Med Año: 2019 Tipo del documento: Article

Texto completo: 1 Base de datos: MEDLINE Asunto principal: Trasplante de Células Madre de Sangre del Cordón Umbilical / Beta-Manosidosis / Beta-Manosidasa Tipo de estudio: Diagnostic_studies Idioma: En Revista: Mol Genet Genomic Med Año: 2019 Tipo del documento: Article