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

doi:10.1007/s10545-017-0103-x

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 journal › Article › peer-review

Original language	English
Pages (from-to)	877-883
Number of pages	7
Journal	Journal of Inherited Metabolic Disease
Volume	41
Issue number	5
DOIs	https://doi.org/10.1007/s10545-017-0103-x
State	Published - Sep 1 2018
Externally published	Yes

ASJC Scopus Subject Areas

Genetics
Genetics(clinical)

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10.1007/s10545-017-0103-x

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Schiffmann, R., Wallace, M. E., Rinaldi, D., Ledoux, I., Luton, M. P., Coleman, S., Akman, H. O., Martin, K., Hogrel, J. Y., Blankenship, D., Turner, J., & Mochel, F. (2018). A double-blind, placebo-controlled trial of triheptanoin in adult polyglucosan body disease and open-label, long-term outcome. Journal of Inherited Metabolic Disease, 41(5), 877-883. https://doi.org/10.1007/s10545-017-0103-x

Schiffmann, R, Wallace, ME, Rinaldi, D, Ledoux, I, Luton, MP, Coleman, S, Akman, HO, Martin, K, Hogrel, JY, Blankenship, D, Turner, J & Mochel, F 2018, 'A double-blind, placebo-controlled trial of triheptanoin in adult polyglucosan body disease and open-label, long-term outcome', Journal of Inherited Metabolic Disease, vol. 41, no. 5, pp. 877-883. https://doi.org/10.1007/s10545-017-0103-x

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AU - Schiffmann, Raphael

AU - Wallace, Mary E.

AU - Rinaldi, Daisy

AU - Ledoux, Isabelle

AU - Luton, Marie Pierre

AU - Coleman, Scott

AU - Akman, H. Orhan

AU - Martin, Karine

AU - Hogrel, Jean Yves

AU - Blankenship, Derek

AU - Turner, Jacob

AU - Mochel, Fanny

PY - 2018/9/1

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SN - 0141-8955

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JO - Journal of Inherited Metabolic Disease

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IS - 5

ER -

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

ASJC Scopus Subject Areas

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