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Lifetime Impact Study for Achondroplasia (LISA): Findings from an observational and multinational study focused on health-related quality of life in individuals with achondroplasia in Latin America

Juan Llerena Jr, Pablo Rosselli, Amanda Aragao, Cristina Valenzuela, Debora Bertola, Yaneth Mendez, Mariana del Pino, Nicolette Calvacanti, Paula Thomazinho, Jeanne M. Pimenta, Shelda Cohen, Tom Butt, Jose C. Thomaz Jr, Renee Shediac , Richard Rowell, Tatiana S.P.C. Magalhaes, Chong Kim, Virginia Fano

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Two-Year Outcomes of Valoctocogene Roxaparvovec Therapy for Hemophilia A

Johnny Mahlangu, M.B., B.Ch., M.Med., Radoslaw Kaczmarek, Ph.D., Annette von Drygalski, M.D., Pharm.D., Susan Shapiro, M.D., Ph.D., Sheng-Chieh Chou, M.D., Margareth C. Ozelo, M.D., Ph.D., Gili Kenet, M.D., Flora Peyvandi, M.D., Ph.D., Michael Wang, M.D., Bella Madan, M.D., Nigel S. Key, M.D., Michael Laffan, D.M., Ph.D., et al., for the GENEr8-1 Trial 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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Diagnosing mucopolysaccharidosis IVA

Timothy C. Wood, Katie Harvey, Michael Beck, Maira Graeff Burin, Yin-Hsiu Chien, Heather J. Church, Vânia D’Almeida, Otto P. van Diggelen, Michael Fietz, Roberto Giugliani, Paul Harmatz, Sara M. Hawley, Wuh-Liang Hwu, David Ketteridge, Zoltan Lukacs, Nicole Miller, Marzia Pasquali, Andrea Schenone, Jerry N. Thompson, Karen Tylee, Chunli Yu, Christian J. Hendriksz