Intrathecal/Intracerebroventricular enzyme replacement therapy for the mucopolysaccharidoses: efficacy, safety, and prospects

Roberto Giugliani, Amauri Dalla Corte, Fabiano Poswar, Cláudia Vanzella, Dafne Horovitz, Mariluce Riegel, Guilherme Baldo, Filippo Vairo
  • Expert Opinion on Orphan Drugs, June 2018, Taylor & Francis
  • DOI: 10.1080/21678707.2018.1487838
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