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
Issue number5
StatePublished - Sep 1 2018
Externally publishedYes

ASJC Scopus Subject Areas

  • Genetics
  • Genetics(clinical)

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