@article{202109.0192, doi = {10.20944/preprints202109.0192.v1}, url = {https://doi.org/10.20944/preprints202109.0192.v1}, year = 2021, month = {September}, publisher = {Preprints}, author = {Roberto Giugliani and Ana Maria Martins and Torayuki Okuyama and Yoshikatsu Eto and Norio Sakai and Kimitoshi Nakamura and Hideto Morimoto and Kohtaro Minami and Tatsuyoshi Yamamoto and Mariko Yamaoka and Toshiaki Ikeda and Sairei So and Kazunori Tanizawa and Hiroyuki Sonoda and Mathias Schmidt and Yuji Sato}, title = {Enzyme Replacement Therapy with Pabinafusp Alfa for Neuronopathic Mucopolysaccharidosis II: an Integrated Analysis of Preclinical and Clinical Data}, journal = {Preprints} }