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Effects of miglustat therapy on neurological disorder and survival in early-infantile Niemann-Pick disease type C: a national French retrospective study.

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Title: Effects of miglustat therapy on neurological disorder and survival in early-infantile Niemann-Pick disease type C: a national French retrospective study.
Authors: Freihuber, Cécile1 (AUTHOR), Dahmani-Rabehi, Bahia2 (AUTHOR), Brassier, Anaïs3 (AUTHOR), Broué, Pierre4 (AUTHOR), Cances, Claude5 (AUTHOR), Chabrol, Brigitte6 (AUTHOR), Eyer, Didier7 (AUTHOR), Labarthe, François8 (AUTHOR), Latour, Philippe9 (AUTHOR), Levade, Thierry10,11 (AUTHOR), Pichard, Samia3 (AUTHOR), Sevin, Caroline12 (AUTHOR), Vanier, Marie T.13,14 (AUTHOR), Héron, Bénédicte1 (AUTHOR) benedicte.heron@aphp.fr
Source: Orphanet Journal of Rare Diseases. 7/21/2023, Vol. 18 Issue 1, p1-12. 12p.
Database: Academic Search Ultimate
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ISSN:17501172
DOI:10.1186/s13023-023-02804-4