A multicenter open-label extension study of intrathecal heparan-N-sulfatase in patients with Sanfilippo syndrome type A

Publication date: Available online 7 July 2021Source: Molecular Genetics and MetabolismAuthor(s): Frits A. Wijburg, Chester B. Whitley, Joseph Muenzer, Serena Gasperini, Mireia del Toro, Nicole Muschol, Maureen Cleary, Caroline Sevin, Elsa Shapiro, David Alexanderian

Source: Molecular Genetics and Metabolism - July 9, 2021 Category: Genetics & Stem Cells Source Type: research