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排名必读论文期刊顶会热点AI 简史溯源树小脉星探人才迁徙塔尖人才国防情报追踪开源工具智慧手语
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P012: Pegtibatinase, an investigational enzyme replacement therapy for the treatment of classical homocystinuria: Latest findings from the COMPOSE phase 1/2 trial
Can Ficicioglu,Jaya Ganesh, Wendy Smith,Melissa Lah, David Kudrow, Jalé Güner, Sharon McDermott, Sagar Vaidya, Liz Wilkening,Janet Thomas, Harvey Levy
Genetics in Medicine Open (2024): 100889
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Pegtibatinase, an investigational enzyme replacement therapy, for the treatment of classical homocystinuria (HCU): Design of the harmony Phase 3 study
Janet Thomas, Tawfeg Ben-Omran, Harvey Levy,Francois Maillot, Sharon McDermott, Sergio Slawka,Can Ficicioglu
Molecular Genetics and Metabolismno. 4 (2024): 108322
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Latest results from the COMPOSE® Phase 1/2 trial for the treatment of classical homocystinuria (HCU) using pegtibatinase, a novel investigational enzyme replacement therapy
Janet Thomas,Jaya Ganesh, Wendy Smith,Melissa Lah, David Kudrow, Jalé Güner, Sharon McDermott, Sagar A. Vaidya, Liz Wilkening, Harvey Levy,Can Ficicioglu
Molecular Genetics and Metabolismno. 4 (2024): 108314
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A review of provider experiences with newborn screening for Krabbe disease in Pennsylvania
Kaylee Williams, Caitlin Menello,Nadene D. Henderson,Nicole Engelhardt, Ashley Lahr, Sarah Schmidt,Can Ficicioglu,Damara Ortiz
Molecular Genetics and Metabolismno. 2 (2023): 107360-107360
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O06: Insights from the first genetic evaluation of a longitudinal natural history study in classical homocystinuria (HCU)*
Tiziano Pramparo,Kimberly Chapman, Ying Chen,Can Ficicioglu,Harvey Levy,Janet Thomas, Sagar Vaidya, Steve Rodems, Ellen Crushell
Genetics in Medicine Openno. 1 (2023): 100007-100007
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