Review of clinical trials and guidelines for children and youth with mucopolysaccharidosis: outcome selection and measurement
粘多糖贮积症儿童和青少年临床试验及指南综述：结局指标的选择和测量
期刊:Orphanet Journal of Rare Diseases
影响因子:3.5
doi:10.1186/s13023-024-03364-x
Howie, Alison H; Tingley, Kylie; Inbar-Feigenberg, Michal; Mitchell, John J; Angel, Kim; Gentle, Jenifer; Smith, Maureen; Offringa, Martin; Butcher, Nancy J; Campeau, Philippe M; Chakraborty, Pranesh; Chan, Alicia; Fergusson, Dean; Mamak, Eva; McClelland, Peyton; Mercimek-Andrews, Saadet; Mhanni, Aizeddin; Moazin, Zeinab; Rockman-Greenberg, Cheryl; Rupar, C Anthony; Skidmore, Becky; Stockler, Sylvia; Thavorn, Kednapa; Wyatt, Alexandra; Potter, Beth K
