TrialOrphanet journal of rare diseases2018

Neurocognitive and somatic stabilization in pediatric patients with severe Mucopolysaccharidosis Type I after 52 weeks of intravenous brain-penetrating insulin receptor antibody-iduronidase fusion protein (valanafusp alpha): an open label phase 1-2 trial.

Roberto Giugliani et al.PubMed ↗Full text ↗Publisher ↗

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