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收藏Pegvaliase therapy for phenylketonuria: Real-world safety, efficacy, and medication access outcomes in a pharmacist-led pegvaliase programPegvaliase疗法治疗苯丙酮尿症:药师主导的Pegvaliase项目中的真实世界安全性、有效性与用药可及性结局
Shrestha, Sofia; Zagel, Alicia L.; Pillai, Nishitha R.; Ahmed, Alia; Jacobson, Jenny; Ranasinghe, Alicia; Whitley, Chester B.; Jarnes, Jeanine R.
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收藏Safety and efficacy of migalastat in adolescent patients with Fabry disease: Results from ASPIRE , a phase 3b, open-label, single-arm, 12-month clinical trial, and its open-label extension
Ramaswami, Uma; Font-Montgomery, Esperanza; Goker-Alpan, Ozlem; Ortiz, Damara; Sanchez-Valle, Amarilis; Whitley, Chester B.; Wilcox, William R.; Jiang, Hai; Lawson, Lee Ann; Vosk, Jennie; Yang, Haichen; Hopkin, Robert J.
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收藏Clinical assessment of disease severity in patients with Fabry disease treated with pegunigalsidase alfa: An integrated analysis
Hughes, Derralynn; Linhart, Ales; Wallace, Eric L.; Wilcox, William R.; Whitley, Chester B.; Vujkovac, Bojan; Koulinska, Irene; Piotti, Giovanni; Chertkoff, Raul; Alon, Sari; Sakov, Anat; Pisani, Antonio; Bernat, John A.
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收藏Long-term safety and efficacy of migalastat in adolescent patients with Fabry disease: Results from the ASPIRE study and open-label extension
Ramaswami, Uma; Font-Montgomery, Esperanza E.; Goker-Alpan, Ozlem; Ortiz, Damara; Sanchez-Valle, Amarilis; Whitley, Chester B.; Wilcox, William R.; Jiang, Hai; Lawson, Lee Ann; Vosk, Jennie; Yang, Haichen; Hopkin, Robert J.
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收藏Isaralgagene civaparvovec (ST-920) gene therapy in adults with Fabry disease: Updated results from an ongoing phase 1/2 study (STAAR)
Hughes, Derralynn; Wilcox, William; Hopkin, Robert J.; Ganesh, Jaya; Bernat, John; Goker-Alpan, Ozlem; Nicholls, Kathy; Deegan, Patrick; Pahl, Madeleine; Whitley, Chester B.; Chen, Michael; Cao, Liching; Schreeb, Katharina H.
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收藏Phenotype differentiation and white matter preservation by early hematopoietic cell transplantation - Free-water diffusion tensor MRI study of the brain in mucopolysaccharidosis type I patients
Nestrasil, Igor; Svatkova, Alena; Pasternak, Ofer; Eisengart, Julie B.; Rudser, Kyle; Mueller, Bryon A.; Bednarik, Petr; Delaney, Kate; Shapiro, Elsa; Whitley, Chester B.
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收藏Quality of life of migalastat-treated adolescents with Fabry disease: Results from the ASPIRE study and open-label extension
Hopkin, Robert J.; Sanchez-Valle, Amarilis; Font-Montgomery, Esperanza E.; Goker-Alpan, Ozlem; Ortiz, Damara; Whitley, chester B.; Wilcox, William R.; Jiang, Hai; Lawson, Lee Ann; Vosk, Jennie; Yang, Haichen; Ramaswami, Uma
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收藏A phase 1/2 study of LY3884961 (PR001) an AAV9-based gene therapy for Gaucher disease type 2-A clinical update from the PROVIDE trial
Neuhaus, Sarah; Tamburri, Paul; Whitley, Chester B.; Jones, Simon A.; Donald, Aimee; Harmatz, Paul; Blair, David R.; Chang, Irene; Gallagher, Renata C.; Rajan, Deepa S.; Goker-Alpan, Ozlem; Beckerman, Yael; Lopez, Victor A.; Hatch, Daniel; Shaughnessy, Lee
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收藏Bone marrow transplantation reverses metabolic alterations in multiple sulfatase deficiency: a case series
Pillai, Nishitha R.; Liu, Ning; Li, Xiyuan; Li, Xiqi; Ahrens-Nicklas, Rebecca; Adang, Laura; Eisengart, Julie B.; Bronken, Grace; Gupta, Ashish; Lund, Troy C.; Whitley, Chester B.; Elsea, Sarah H.; Orchard, Paul J.
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