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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 by Can Ficicioglu, Jaya Ganesh, Wendy Smith, Melissa Lah, David Kudrow, Jalé Güner, Sharon McDermott, Sagar Vaidya, Liz Wilkening, Janet Thomas, Harvey Levy
Published 2024-01-01
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P001: Outcomes In 14 live births resulting from pegvaliase-treated pregnancies in females with PKU* by Caide Bier, Kaelin Dickey, Hans Andersson, Markey McNutt, Danielle Vice, Erin Cooney, Megan Morand, Joseph Ray, Rebecca Sponberg, Richard Chang, Monica Boyer, Brittan Bibb, Angela Crutcher, Melissa Lah, Laura Davis-Keppen, Cindy Matthes
Published 2024-01-01
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O38: The impact of whole genome sequencing in a diverse global population of genetic disease patients* by Ryan Taft, Erin Thorpe, John Belmont, Taylor Williams, Chad Shaw, Jason Button, Julia Ortega, Keisha Robinson, Marilyn Jones, Diane Masser-Frye, Donald Basel, Chester Brown, Keith Vaux, Aime Lumaka, Fabio Sirchia, Milagros Dueñas Roque, Mario Cornejo-Olivas, Jeny Bazalar-Montoya, Nora Urraca, Alejandra Salguero, Samuel Wiafe, Romina Foster-Bonds, Erin Royer, Michelle Gallas, Pilar Magoulas, Adeline Vanderver, Marwan Shinawi, Alan Taylor, Kristen Fishler, Duncan Henry, Daria Salyakina, Kate Gibson, Melissa Lah, Alka Malhotra, James Avecilla, Andrew Warren, Denise Perry, Max Arseneault
Published 2023-01-01
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