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A double-blind, placebo-controlled trial of triheptanoin in adult polyglucosan body disease and open-label, long-term outcome

  • Raphael Schiffmann
  • , Mary E. Wallace
  • , Daisy Rinaldi
  • , Isabelle Ledoux
  • , Marie Pierre Luton
  • , Scott Coleman
  • , H. Orhan Akman
  • , Karine Martin
  • , Jean Yves Hogrel
  • , Derek Blankenship
  • , Jacob Turner
  • , Fanny Mochel

Research output: Contribution to journalArticlepeer-review

Original languageEnglish
Pages (from-to)877-883
Number of pages7
JournalJournal of Inherited Metabolic Disease
Volume41
Issue number5
DOIs
StatePublished - Sep 1 2018
Externally publishedYes

ASJC Scopus Subject Areas

  • Genetics
  • Genetics(clinical)

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