A phase 2 clinical study in Niemann-Pick disease type C and GM2 gangliosidosis patients (RAINBOW)

被引：0
|
作者：
Giugliani, Roberto [1 ]
Valle, Daniel A. D. [2 ]
Horovitz, Dafne [3 ]
Martins, Ana Maria [4 ]
Crapard, Laetitia [5 ]
Doppler, Emilie [5 ]
Hurst, Noemie [5 ]
de Frutos, Laura Lopez [5 ]
Luzy, Cecile Paquet [5 ]
机构：
[1] Univ Fed Rio Grande do Sul, Porto Alegre, RS, Brazil
[2] Hosp Pequeno Principe, Curitiba, Parana, Brazil
[3] Inst Fernandes Figueira Fiocruz, Rio De Janeiro, Brazil
[4] Fede Univ Sao Paulo, Sao Paulo, Brazil
[5] 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.2023.107847
中图分类号：
R5 [内科学];
学科分类号：
1002 ; 100201 ;
摘要：
108
引用
收藏
页数：2
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