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A link between baseline neurofilament light chain and primary substrate accumulation in cerebrospinal fluid, and clinical outcomes in patients with MPS II from a phase 2/3 clinical trial and extension study of intrathecal idursulfase Argueta, Christian; Brekk, Oeystein Roed; Wang, Scarlett; Feng, Qihua; Ahmed, Mariam; Mcdonnell, Scott R. P.; Pan, Luying; Plavina, Tatiana; Whiteman, David A. H. 分享 收藏
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Neutralizing Antibody Sample Testing and Report Harmonization (vol 26, 80, 2024) Jani, Darshana; Gunsior, Michele; Marsden, Robin; Cowan, Kyra J.; Irvin, Susan C.; Hay, Laura Schild; Ward, Bethany; Armstrong, Luke; Azadeh, Mitra; Cao, Liching; Carmean, Rebecca; Delcarpini, Jason; Dholakiya, Sanjay L.; Hays, Amanda; Hosback, Sarah; Hu, Zheng; Kulagina, Nadia; Kumar, Seema; Lai, Ching Ha; Lichtfuss, Marit; Liu, Hsing-Yin; Liu, Susana; Mozaffari, Reza; Pan, Luying; Pennucci, Jason; Poupart, Marie-Eve; Saini, Gurleen; Snoeck, Veerle; Storey, Kristine; Turner, Amy; Vainshtein, Inna; Verthelyi, Daniela; Wala, Iwona; Yang, Lili; Yang, Lin 分享 收藏
Neutralizing Antibody Sample Testing and Report Harmonization Jani, Darshana; Gunsior, Michele; Marsden, Robin; Cowan, Kyra J.; Irvin, Susan C.; Hay, Laura Schild; Ward, Bethany; Armstrong, Luke; Azadeh, Mitra; Cao, Liching; Carmean, Rebecca; DelCarpini, Jason; Dholakiya, Sanjay L.; Hays, Amanda; Hosback, Sarah; Hu, Zheng; Kulagina, Nadia; Kumar, Seema; Lai, Ching Ha; Lichtfuss, Marit; Liu, Hsing-Yin; Liu, Susana; Mozaffari, Reza; Pan, Luying; Pennucci, Jason; Poupart, Marie-Eve; Saini, Gurleen; Snoeck, Veerle; Storey, Kristine; Turner, Amy; Vainshtein, Inna; Verthelyi, Daniela; Wala, Iwona; Yang, Lili; Yang, Lin 分享 收藏
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Anti-drug Antibody Sample Testing and Reporting Harmonization Jani, Darshana; Marsden, Robin; Gunsior, Michele; Hay, Laura Schild; Ward, Bethany; Cowan, Kyra J.; Azadeh, Mitra; Barker, Breann; Cao, Liching; Closson, Kristin R.; Coble, Kelly; Dholakiya, Sanjay L.; Dusseault, Julie; Hays, Amanda; Herl, Carina; Hodsdon, Michael E.; Irvin, Susan C.; Kirshner, Susan; Kolaitis, Gerry; Kulagina, Nadia; Kumar, Seema; Lai, Ching Ha; Lipari, Francesco; Liu, Susana; Merdek, Keith D.; Moldovan, Ioana R.; Mozaffari, Reza; Pan, Luying; Place, Corina; Snoeck, Veerle; Manning, Marta Starcevic; Stocker, Dennis; Tary-Lehmann, Magdalena; Turner, Amy; Vainshtein, Inna; Verthelyi, Daniela; Williams, William T.; Yan, Haoheng; Yan, Weili; Yang, Lili; Yang, Lin; Zemo, Jennifer; Zhong, Zhandong Don 分享 收藏
A long-term extension study evaluating intrathecal idursulfase-IT in children with Hunter syndrome and cognitive impairment Muenzer, Joseph; Hendriksz, Christian J.; Stein, Margot B.; Fan, Zheng; Kearney, Shauna; Horton, Johan; Vijayaraghavan, Suresh; Santra, Saikat; Solanki, Guirish A.; Pan, Luying; Alexanderian, David 分享 收藏
A phase 1/2 study of intrathecal heparan-N-sulfatase in patients with mucopolysaccharidosis IIIA Jones, Simon A.; Breen, Catherine; Heap, Fiona; Rust, Stewart; de Ruijter, Jessica; Tump, Evelien; Marchal, Jan Pieter; Pan, Luying; Qiu, Yongchang; Chung, Jou-Ku; Nair, Nitin; Haslett, Patrick A. J.; Barbier, Ann J.; Wijburg, Frits A. 分享 收藏
A phase I/II study of intrathecal idursulfase-IT in children with severe mucopolysaccharidosis II Muenzer, Joseph; Hendriksz, Christian J.; Fan, Zheng; Vijayaraghavan, Suresh; Perry, Victor; Santra, Saikat; Solanki, Guirish A.; Mascelli, Mary Ann; Pan, Luying; Wang, Nan; Sciarappa, Kenneth; Barbier, Ann J. 分享 收藏
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Sulfatide levels correlate with severity of neuropathy in metachromatic leukodystrophy Dali, Christine I.; Barton, Norman W.; Farah, Mohamed H.; Moldovan, Mihai; Mansson, Jan-Eric; Nair, Nitin; Duno, Morten; Risom, Lotte; Cao, Hongmei; Pan, Luying; Sellos-Moura, Marcia; Corse, Andrea M.; Krarup, Christian 分享 收藏
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Long-term biomarker and cognitive follow-up of children with Hunter syndrome receiving intrathecal enzyme replacement therapy Muenzer, Joseph; Hendriksz, Christian J.; Stein, Margot B.; Fan, Zheng; Kearney, Shauna; Horton, Johan; Vijayaraghavan, Suresh; Santra, Saikat; Solanki, Guirish A.; Pan, Luying; Wang, Nan; Mascelli, Mary Ann; Sciarappa, Kenneth; Barbier, Ann J. 分享 收藏
Investigational intrathecal (IT) enzyme replacement therapy for the severe form of Hunter syndrome (mucopolysaccharidosis type II, MPS II) Muenzer, Joseph; Hendriksz, Christian J.; Stein, Margot B.; Fan, Zheng; Kearney, Shauna; Horton, Johan; Vijayaraghavan, Suresh; Perry, Victor; Santra, Saikat; Solanki, Guirish A.; Wang, Nan; Mascelli, Mary Ann; Pan, Luying; Sciarapp, Kenneth; Barbier, Ann J. 分享 收藏
Antigenic differences in patients receiving velaglucerase alfa or imiglucerase treatment Ruiz, Joan; Barzegar, Samad; Clarke, Andrea; Durant, Judith; Milhaven, John; Nadeau, Marie; Nguyen, Thu; Oommen, Susan; Pan, Luying; Rabinovich, Deana; Rickenbach, Brett; Sllos-Moura, Mrcia; Shi, Philip 分享 收藏
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