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Response to triheptanoin therapy in critically ill patients with LC-FAOD: Report of patients treated through an expanded access program.
Vockley, Jerry; Enns, Gregory M; Ramirez, Antonio Nino; Bedrosian, Camille L; Reineking, Bridget; Lu, Xiaoxiao; Ray, Kathryn; Rahman, Syeda; Marsden, Deborah.
Affiliation
  • Vockley J; UPMC Children's Hospital of Pittsburgh, Pittsburgh, PA, United States of America. Electronic address: gerard.vockley@chp.edu.
  • Enns GM; Stanford University, Division of Medical Genetics, Palo Alto, CA, United States of America.
  • Ramirez AN; Ultragenyx Pharmaceutical Inc., Novato, CA, United States of America.
  • Bedrosian CL; Ultragenyx Pharmaceutical Inc., Novato, CA, United States of America.
  • Reineking B; Ultragenyx Pharmaceutical Inc., Novato, CA, United States of America.
  • Lu X; Ultragenyx Pharmaceutical Inc., Novato, CA, United States of America.
  • Ray K; Ultragenyx Pharmaceutical Inc., Novato, CA, United States of America.
  • Rahman S; Ultragenyx Pharmaceutical Inc., Novato, CA, United States of America.
  • Marsden D; Ultragenyx Pharmaceutical Inc., Novato, CA, United States of America.
Mol Genet Metab ; 136(2): 152-162, 2022 06.
Article in En | MEDLINE | ID: mdl-35459555
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Full text: 1 Database: MEDLINE Main subject: Lipid Metabolism, Inborn Errors / Cardiomyopathies Type of study: Observational_studies Limits: Humans / Infant Language: En Year: 2022 Type: Article
Fulltext
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Full text: 1 Database: MEDLINE Main subject: Lipid Metabolism, Inborn Errors / Cardiomyopathies Type of study: Observational_studies Limits: Humans / Infant Language: En Year: 2022 Type: Article