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O6-2 Cerebral PET and disability evaluation in patients with Niemann Pick disease Type C treated with miglustat

✍ Scribed by M.S. Perez Poyato; M. Pineda Marfa; M.A. Vilaseca; M.M. O'Callaghan Gordo; J. Macias; M.J. Coll


Book ID
114359851
Publisher
Elsevier Science
Year
2009
Tongue
English
Weight
43 KB
Volume
13
Category
Article
ISSN
1090-3798

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## Abstract Niemann–Pick disease type C (NPC) is a progressive neurodegenerative disorder for which there is no effective treatment other than supportive therapy. Recently, the oral medication miglustat has been offered as a possible therapy aimed at reducing pathological substrate accumulation. Th