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Adult Niemann-Pick disease type C in France: clinical phenotypes and long-term miglustat treatment effect.

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Title: Adult Niemann-Pick disease type C in France: clinical phenotypes and long-term miglustat treatment effect.
Authors: Nadjar, Yann1 yann.nadjar@aphp.fr, Hütter-Moncada, Ana Lucia2 drmoncadaguifarro@gmail.com, Latour, Philippe3 philippe.latour@chu-lyon.fr, Ayrignac, Xavier4 xavier.ayrignac@yahoo.fr, Kaphan, Elsa5 Elsa.KAPHAN@ap-hm.fr, Tranchant, Christine6,7,8 christine.tranchant@chru-strasbourg.fr, Cintas, Pascal9 cintas.p@chu-toulouse.fr, Degardin, Adrian10 adriandegardin@msn.com, Goizet, Cyril11 cyril.goizet@chu-bordeaux.fr, Laurencin, Chloe12 clo.laurencin@gmail.com, Martzolff, Lionel13 martzolffl@ghrmsa.fr, Tilikete, Caroline14 caroline.tilikete@chu-lyon.fr, Anheim, Mathieu6,7,8 mathieu.anheim@chru-strasbourg.fr, Audoin, Bertrand15,16 bertrand.audoin@ap-hm.fr, Deramecourt, Vincent17 vincent.deramecourt@chru-lille.fr, De Gaillarbois, Thierry Dubard18 thierry.dubard@orange.fr, Roze, Emmanuel1,19 emmanuel.flamand-roze@aphp.fr, Lamari, Foudil20 foudil.lamari@ap.hp.fr, Vanier, Marie T.21,22 vaniermtv@gmail.com, Héron, Bénédicte23 benedicte.heron@aphp.fr
Source: Orphanet Journal of Rare Diseases. 10/1/2018, Vol. 13 Issue 1, pN.PAG-N.PAG. 1p.
Database: Academic Search Ultimate
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ISSN:17501172
DOI:10.1186/s13023-018-0913-4