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Caregiver Perspectives on Patients with Mucopolysaccharidosis II Treated with Pabinafusp Alfa: Results of Qualitative Interviews in Japan

2023-09-01

Abstract excerpt

<title>Abstract</title> <p>Background Mucopolysaccharidosis type II (MPS II), or Hunter syndrome, is a rare inherited X-linked metabolic disorder predominantly affecting males. Pabinafusp alfa, an iduronate-2-sulfatase enzyme that can cross the blood-brain barrier, was approved in Japan in 2021 for use in the first enzyme replacement therapy targeting both the neuropathic and somatic symptoms of MPS II. This stu...

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Literature Corpus work: 91bd0156-1dec-532d-8c17-d137bbff66df
DOI: 10.21203/rs.3.rs-3274135/v1

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