Partial suppression of BCAA catabolism as a potential therapy for BCKDK deficiency by Laura Ohl, Amanda Kuhs, Ryan Pluck, Emily Durham, Michael Noji, Nathan D. Philip, Zoltan Arany, Rebecca C. Ahrens-Nicklas

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Rapid and definitive treatment of phenylketonuria in variant-humanized mice with corrective editing by Dominique L. Brooks, Manuel J. Carrasco, Ping Qu, William H. Peranteau, Rebecca C. Ahrens-Nicklas, Kiran Musunuru, Mohamad-Gabriel Alameh, Xiao Wang

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Characterizing asparagine synthetase deficiency variants in lymphoblastoid cell lines by Stephen J. Staklinski, Mario C. Chang, Rebecca C. Ahrens‐Nicklas, Shagun Kaur, Arianna K. Stefanatos, Elizabeth E. Dudenhausen, Matthew E. Merritt, Michael S. Kilberg

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The Importance of Succinylacetone: Tyrosinemia Type I Presenting with Hyperinsulinism and Multiorgan Failure Following Normal Newborn Screening by Jessica R. C. Priestley, Hana Alharbi, Katharine Press Callahan, Herodes Guzman, Irma Payan-Walters, Ligia Smith, Can Ficicioglu, Rebecca D. Ganetzky, Rebecca C. Ahrens-Nicklas

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Bone marrow transplantation increases sulfatase activity in somatic tissues in a multiple sulfatase deficiency mouse model by Maximiliano Presa, Vi Pham, Somdatta Ray, Pierre-Alexandre Piec, Jennifer Ryan, Timothy Billings, Harold Coombs, Lars Schlotawa, Troy Lund, Rebecca C. Ahrens-Nicklas, Cathleen Lutz

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Newborn Screening for Pompe Disease: Pennsylvania Experience by Can Ficicioglu, Rebecca C. Ahrens-Nicklas, Joshua Barch, Sanmati R. Cuddapah, Brenda S. DiBoscio, James C. DiPerna, Patricia L. Gordon, Nadene Henderson, Caitlin Menello, Nicole Luongo, Damara Ortiz, Rui Xiao

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Newborn Screening for X-Linked Adrenoleukodystrophy: Review of Data and Outcomes in Pennsylvania by Jessica R. C. Priestley, Laura A. Adang, Sarah Drewes Williams, Uta Lichter-Konecki, Caitlin Menello, Nicole M. Engelhardt, James C. DiPerna, Brenda DiBoscio, Rebecca C. Ahrens-Nicklas, Andrew C. Edmondson, Francis Jeshira Reynoso Santos, Can Ficicioglu

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Drug screening identifies tazarotene and bexarotene as therapeutic agents in multiple sulfatase deficiency by Lars Schlotawa, Karolina Tyka, Matthias Kettwig, Rebecca C Ahrens‐Nicklas, Matthias Baud, Tea Berulava, Nicola Brunetti‐Pierri, Alyssa Gagne, Zackary M Herbst, Jean A Maguire, Jlenia Monfregola, Tonatiuh Pena, Karthikeyan Radhakrishnan, Sophie Schröder, Elisa A Waxman, Andrea Ballabio, Thomas Dierks, André Fischer, Deborah L French, Michael H Gelb, Jutta Gärtner

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