Doenças raras na infância: terapia enzimática inovadora no tratamento da Síndrome de Hunter mostra avanços no tratamento ao sistema nervoso central (SNC)

Referências

1. Muenzer J, Ho C, Lau H, et al. Community consensus for heparan sulfate as a biomarker to support accelerated approval in neuronopathic mucopolysaccharidoses. Mol Genet Metab. 2024;142(4):108535. doi: 10.1016/j.ymgme.2024.108535. 

2. Muenzer J, Harmatz P, Burton BK, et al. Interim analysis of key clinical outcomes from a phase 1/2 study of weekly intravenous DNL310 (brain-penetrant enzyme replacement therapy) in MPS II. Presented at the 19th Annual WORLD Symposium; 2023 Feb 22–26; Orlando, FL.