Evaluation of AAV vectors with tissue-specific or ubiquitous promoters in a mouse model of mucopolysaccharidosis type IVA by Shaukat A. Khan, Jose Victor Álvarez, F.N.U. Nidhi, Eliana Benincore-Florez, Shunji Tomatsu

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Long-term outcomes of enzyme replacement therapy from a large cohort of Korean patients with mucopolysaccharidosis IVA (Morquio A syndrome) by Juyoung Sung, Insung Kim, Minji Im, Yoon Ji Ahn, Sang-Mi Kim, Ja-Hyun Jang, Hyung-Doo Park, Tae Yeon Jeon, Kyung Rae Ko, Se-Jun Park, Jun Hwa Lee, Eun Young Kim, Chong Kun Cheon, Eungu Kang, Jung-eun Moon, Young Bae Sohn, Hsiang-Yu Lin, Chih-Kuang Chuang, Shuan-Pei Lin, Sung Yoon Cho

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Mucopolysaccharidosis type IVA and severe hidradenitis suppurativa: A case series by Jorga Fialová, MD, Martina Kojanová, MD, PhD, Robert Šáhó, MD, Martin Magner, MD, PhD

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In vivo direct lentiviral gene therapy improves disease pathology in a mucopolysaccharidosis IVA murine model by Betul Celik, Estera Rintz, Shaukat Khan, Andrés Felipe Leal, Fnu Nidhi, Shunji Tomatsu

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