A natural history study of late-infantile and juvenile GM1 and GM2 gangliosidosis (PRONTO): Evaluation of different assessments

被引：0

作者：

Giugliani, Roberto ^{[1
]}

Harmatz, Paul ^{[2
]}

Heron, Benedicte ^{[3
]}

Patterson, Marc ^{[4
]}

Schneider, Susanne ^{[5
]}

Bourchany, Aurelie ^{[6
]}

Hahn, Andreas ^{[7
]}

Valle, Daniel A. D. ^{[8
]}

Barone, Rita Maria Elisa ^{[9
]}

Chabrol, Brigitte ^{[10
]}

Ardissone, Anna ^{[11
]}

Batzios, Spyros ^{[12
]}

Scarpa, Maurizio ^{[13
]}

Crapard, Laetitia ^{[14
]}

de Frutos, Laura Lopez ^{[14
]}

Quintela, Renata Medinaceli ^{[14
]}

Thiers, Aurelien ^{[14
]}

Luzy, Cecile Paquet ^{[14
]}

机构：

[1] Univ Fed Rio Grande do Sul, Porto Alegre, RS, Brazil

[2] Univ Calif San Francisco, Benioff Childrens Hosp, Oakland, CA USA

[3] Armand Trousseau La Roche Guyon Hosp, Paris, France

[4] Mayo Clin, Rochester, MN USA

[5] Ludwig Maximilians Univ Munchen, Munich, Germany

[6] CHU Toulouse, Hosp Enfants, Toulouse, France

[7] Justus Liebig Univ, Giessen, Germany

[8] Hosp Pequeno Principe, Curitiba, Brazil

[9] Presidio Osped Gaspare Rodolico, Catania, Italy

[10] CHU Marseille, La Timone Hosp, Marseille, France

[11] Fdn IRCCS Ist Neurol Besta, Milan, Italy

[12] Great Ormond St Hosp Sick Children, London, England

[13] Univ Hosp Udine, Udine, Italy

[14] Azafaros BV, Naarden, Netherlands

来源：

MOLECULAR GENETICS AND METABOLISM | 2024年 / 141卷 / 02期

关键词：

D O I：

10.1016/j.ymgme.2023.107850

中图分类号：

R5 [内科学];

学科分类号：

1002 ; 100201 ;

摘要：

111

引用

页数：1

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