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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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  Data: 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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  Data: <searchLink fieldCode="JN" term="%22Orphanet+Journal+of+Rare+Diseases%22">Orphanet Journal of Rare Diseases</searchLink>. 7/21/2023, Vol. 18 Issue 1, p1-12. 12p.
PLink https://search.ebscohost.com/login.aspx?direct=true&site=eds-live&db=asn&AN=165465425
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        Value: 10.1186/s13023-023-02804-4
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        Text: English
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              Text: 7/21/2023
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