24 month-treatment with miglustat of three patients with Niemann-Pick disease type C: Follow up using brain spectroscopy

Niemann-Pick C (NPC) is a fatal progressive neurolipidosis. Miglustat, an inhibitor of glycosphingolipid synthesis, has been proposed to treat patients but questions remain regarding its efficacy. A major problem has been the lack of suitable objective efficacy endpoints. Three adults with NPC were...

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Veröffentlicht in:	Molecular genetics and metabolism 2009-02, Vol.96 (2), p.55-58
Hauptverfasser:	Galanaud, Damien, Tourbah, Ayman, Lehéricy, Stéphane, Leveque, Nathalie, Heron, Bénédicte, Billette de Villemeur, Thierry, Guffon, Nathalie, Feillet, François, Baumann, Nicole, Vanier, Marie T., Sedel, Frédéric
Format:	Artikel
Sprache:	eng
Schlagworte:	1-Deoxynojirimycin - analogs & derivatives 1-Deoxynojirimycin - therapeutic use Adult Brain - metabolism Brain - pathology Choline - metabolism Cohort Studies Computer Science Creatine - metabolism Female Humans Inborn errors of metabolism Inherited metabolic diseases Magnetic Resonance Imaging Magnetic resonance spectroscopy Magnetic Resonance Spectroscopy - methods Male Medical Imaging Miglustat Niemann-Pick C Niemann-Pick Disease, Type C - drug therapy Substrate reduction therapy Treatment Outcome Zavesca
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