Caregiver experiences and observations of intrathecal idursulfase-IT treatment in a phase 2/3 trial in pediatric patients with neuronopathic mucopolysaccharidosis II
Article in Orphanet Journal of Rare Diseases (March 2024)
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Article in Orphanet Journal of Rare Diseases (March 2024)
Article in Molecular Therapy (February 2024)
Book chapter (January 2024)