Roberto Giugliani, P. Harmatz, Bénédicte Héron, Marc Patterson, Susanne A Schneider, Aurélie Bourchany, Andreas Hahn, Daniel A.D. Valle, Rita Maria Elisa Barone, Brigitte Chabrol, Anna Ardissone, S. Batzios, Maurizio Scarpa, Nicoleta Carp, Laetitia Crapard, L. L. de Frutos, R. M. Quintela, Aurelien Thiers, C. Luzy
{"title":"A natural history study of late-infantile and juvenile GM1 and GM2 gangliosidoses (PRONTO): Patients' and caregivers' assessments","authors":"Roberto Giugliani, P. Harmatz, Bénédicte Héron, Marc Patterson, Susanne A Schneider, Aurélie Bourchany, Andreas Hahn, Daniel A.D. Valle, Rita Maria Elisa Barone, Brigitte Chabrol, Anna Ardissone, S. Batzios, Maurizio Scarpa, Nicoleta Carp, Laetitia Crapard, L. L. de Frutos, R. M. Quintela, Aurelien Thiers, C. Luzy","doi":"10.1016/j.ymgme.2023.107849","DOIUrl":null,"url":null,"abstract":"","PeriodicalId":504050,"journal":{"name":"Molecular Genetics and Metabolism","volume":"103 1","pages":""},"PeriodicalIF":0.0000,"publicationDate":"2024-02-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":"0","resultStr":null,"platform":"Semanticscholar","paperid":null,"PeriodicalName":"Molecular Genetics and Metabolism","FirstCategoryId":"1085","ListUrlMain":"https://doi.org/10.1016/j.ymgme.2023.107849","RegionNum":0,"RegionCategory":null,"ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":null,"EPubDate":"","PubModel":"","JCR":"","JCRName":"","Score":null,"Total":0}
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