Analysis of caregiver perspectives on patients with mucopolysaccharidosis II treated with pabinafusp alfa: results of qualitative interviews in Japan

Abstract Background Mucopolysaccharidosis type II (MPS II), or Hunter syndrome, is a rare X-linked metabolic disorder predominantly affecting males. Pabinafusp alfa, an iduronate-2-sulfatase enzyme designed to cross the blood-brain barrier, was approved in Japan in 2021 as the first enzyme replaceme...

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Päätekijät:	Kimitoshi Nakamura, Norio Sakai, Mohammad Arif Hossain, Julie B Eisengart, Tatsuyoshi Yamamoto, Kazunori Tanizawa, Sairei So, Mathias Schmidt, Yuji Sato
Aineistotyyppi:	Artigo
Kieli:	Inglês
Julkaistu:	BMC 2024-03-01
Sarja:	Orphanet Journal of Rare Diseases
Aiheet:	Mucopolysaccharidosis type II Hunter syndrome Caregiver-reported outcomes Pabinafusp alfa Treatment experience Quality of life
Linkit:	https://doi.org/10.1186/s13023-024-03112-1
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