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Study of Intraventricular Cerliponase Alfa for CLN2 Disease

Angela Schulz, M.D., Temitayo Ajayi, M.D., Nicola Specchio, M.D., Ph.D., Emily de Los Reyes, M.D., Paul Gissen, M.B., Ch.B., Ph.D., Douglas Ballon, Ph.D., Jonathan P. Dyke, Ph.D., Heather Cahan, M.D., Peter Slasor, Sc.D., David Jacoby, M.D., Ph.D., and Alfried Kohlschütter, M.D., for the CLN2 Study Group

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Intracerebroventricular Cerliponase Alfa for Neuronal Ceroid Lipofuscinosis Type 2 Disease: Clinical Practice Considerations From US Clinics

Emily de los Reyes, MD, Lenora Lehwald, MD, Erika F. Augustine, MD, MS, Elizabeth Berry-Kravis, MD, PhD, Karen Butler, BSN, RN, Natalie Cormier, MSN, Scott Demarest, MD, Sam Lu, MD, Jacqueline Madden, RN, PNP, Joffre Olaya, MD, Susan See, MSN, Amy Vierhile, DNP, RN, James W. Wheless, BPharm, MD, Amy Yang, MD, Jessica Cohen-Pfeffer, MD, Dorna Chu, PharmD, Fernanda Leal-Pardinas, MD, Raymond Y. Wang, MD

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Management Strategies for CLN2 Disease

Ruth E. Williams DM, FRCPCH, Heather R. Adams PhD, Martin Blohm MD, Jessica L. Cohen-Pfeffer MD, Emily de los Reyes MD, Jonas Denecke MD, Kristen Drago RN, CHPPN, Charlie Fairhurst MBBS, FRCPCH, Margie Frazier PhD, Norberto Guelbert MD, PhD, Szilárd Kiss MD, Annamaria Kofler PT, John A. Lawson BMed, FRACP, PhD, Lenora Lehwald MD, Mary-Anne Leung SRD, Svetlana, Mikhaylova MD, Jonathan W. Mink MD, PhD, Miriam Nickel MD, Renée Shediac PhD, Katherine Sims MD, Nicola Specchio MD, PhD, Meral Topcu MD, Ina von Löbbecke PT, Andrea West MSc, Boris Zernikow MD, PhD, Angela Schulz MD, PhD

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Safety and efficacy of cerliponase alfa in children with neuronal ceroid lipofuscinosis type 2 (CLN2 disease): an open-label extension study

Angela Schulz, MD PhD , Nicola Specchio, MD PhD, Emily de los Reyes, MD, Paul Gissen, MD PhD, Miriam Nickel, MD, Marina Trivisano, MD PhD, Shawn C Aylward, MD, Anupam Chakrapani, MD, Christoph Schwering, MD, Eva Wibbeler, MD, Lena Marie Westermann, MD, Douglas J Ballon, PhD, Jonathan P Dyke, PhD DABMP, Anu Cherukuri, PhD, Shailesh Bondade, MD, Peter Slasor, ScD, Jessica Cohen Pfeffer, MD

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Management of early treated adolescents and young adults with phenylketonuria: Development of international consensus recommendations using a modified Delphi approach

Barbara K. Burton, Álvaro Hermida, Amaya Bélanger-Quintana, Heather Bell, Kendra J. Bjoraker, Shawn E. Christ, Mitzie L. Grant, Cary O. Harding, Stephan C.J. Huijbregts, Nicola Longo, Markey C. McNutt II, Mina D. Nguyen-Driver, André L. Santos Pessoa, Júlio César Rocha, Stephanie Sacharow, Amarilis Sanchez-Valle, H. Serap Sivri, Jerry Vockley, Mark Walterfang, Sarah Whittle, Ania C. Muntau