Long-term safety and clinical outcomes of intrathecal heparan-N-sulfatase in patients with Sanfilippo syndrome type A.
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F. Wijburg | S. Rust | E. Shapiro | E. Tump | D. Alexanderian | Simon A. Jones | J. de Ruijter | J. P. Marchal | I. Nestrašil | Fiona Heap
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