Neurodevelopmental status and adaptive behavior of pediatric patients with mucopolysaccharidosis II: a longitudinal observational study

por Muenzer, Joseph, Burton, Barbara K., Amartino, Hernan M., Harmatz, Paul R., Gutiérrez-Solana, Luis González, Ruiz-Garcia, Matilde, Wu, Yuna, Merberg, David, Alexanderian, David, Jones, Simon A.
Publicado 2023

Variables affecting outcomes after allogeneic hematopoietic stem cell transplant for cerebral adrenoleukodystrophy

por Chiesa, Robert, Boelens, Jaap Jan, Duncan, Christine N., Kühl, Jörn-Sven, Sevin, Caroline, Kapoor, Neena, Prasad, Vinod K., Lindemans, Caroline A., Jones, Simon A., Amartino, Hernan M., Algeri, Mattia, Bunin, Nancy, Diaz-de-Heredia, Cristina, Loes, Daniel J., Shamir, Esther, Timm, Alison, McNeil, Elizabeth, Dietz, Andrew C., Orchard, Paul J.
Publicado 2022