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P015: Clinical characterization of classical homocystinuria due to cystathionine-beta synthase deficiency: Results from the ACAPPELLA study by Can Ficicioglu, Melissa Lah, Harvey Levy, Hong Li, Clint Lovell, Cheryl Wong Po Foo, Ellen Crushell, Reena Sharma, Janet Thomas
Published 2025-01-01Get full text
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Lessons learned from 5 years of pegvaliase in US clinics: A case series by Erin Cooney, Zineb Ammous, Tricia Bender, Gillian E. Clague, Marilyn Clifford, Angela Crutcher, Laura Davis-Keppen, Kirsten Havens, Melissa Lah, Stephanie Sacharow, Amarilis Sanchez-Valle, Erika Vucko, Bridget Wardley, Leah Wessenberg, Hans C. Andersson
Published 2025-03-01Get full text
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P047: Interim results from the APHENITY extension study: Sepiapterin reduces blood Phe with improved dietary Phe tolerance in participants with phenylketonuria by Nicola Longo, Francjan van Spronsen, Ania Muntau, Lali Margvelashvili, Heidi Peters, Ida Vanessa Schwartz, Takashi Hamazaki, Maria Avanise Yumi Minami, Laura Guilder, Drago Bratkovic, Melissa Lah, Janet Thomas, Frank Rutsch, Ertugrul Kiykim, Roberto Zori, Mika Ishige, Kimberly Ingalls, Catalina Hughes, Lan Gao, Zhenming Zhao, Neil Smith
Published 2025-01-01Get full text
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