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

yann Nadjar 1 Ana Lucia Hütter-Moncada 2 Philippe Latour 3 Xavier Ayrignac 4 Elsa Kaphan 5 Christine Tranchant 6, 7, 8 Pascal Cintas 9 Adrian Degardin 10 Cyril Goizet 11, 12 Chloe Laurencin 13 Lionel Martzolff 14 Caroline Tilikete 15, 16 Mathieu Anheim 17, 7, 8 Bertrand Audoin 18, 19, 20 Vincent Deramecourt 21 Thierry Dubard de Gaillarbois 22 Emmanuel Roze 1, 23 Foudil Lamari 24 Marie Vanier 25, 26 Bénédicte Héron 27, 28 
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https://hal-amu.archives-ouvertes.fr/hal-03541909
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Submitted on : Tuesday, January 25, 2022 - 8:33:11 AM
Last modification on : Tuesday, May 3, 2022 - 3:32:33 AM

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yann Nadjar, Ana Lucia Hütter-Moncada, Philippe Latour, Xavier Ayrignac, Elsa Kaphan, et al.. Adult Niemann-Pick disease type C in France: clinical phenotypes and long-term miglustat treatment effect. Orphanet Journal of Rare Diseases, BioMed Central, 2018, 13 (1), pp.175. ⟨10.1186/s13023-018-0913-4⟩. ⟨hal-03541909⟩

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