Intrathecally administered recombinant human arylsulfatase A in patients with late-infantile metachromatic leukodystrophy: Phase 2b clinical trial design

Whiteman, David A.H.; Vanderver, Adeline; Krägeloh‐Mann, Ingeborg; Gray, Jill; Wu, James X.; Wasilewski, Margaret

doi:10.1016/j.ymgme.2018.12.394

Molecular Genetics and Metabolism

2019

DOI: 10.1016/j.ymgme.2018.12.394

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Intrathecally administered recombinant human arylsulfatase A in patients with late-infantile metachromatic leukodystrophy: Phase 2b clinical trial design

David A.H. Whiteman

Adeline Vanderver

Ingeborg Krägeloh‐Mann

et al.

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Intrathecally administered recombinant human arylsulfatase A in patients with late-infantile metachromatic leukodystrophy: Phase 2b clinical trial design

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