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C. Greg Guyer

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Marni Kottle

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Brian R. Mueller

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Amy Wireman

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Alexander Hardy

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Elizabeth McKee Anderson

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Robert J. Hombach

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Richard A. Meier

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David E.I. Pyott

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Global Compliance and Ethics Program

• Publication

Achondroplasia: a comprehensive clinical review

Richard M. Pauli

• Publication

Lifetime impact of achondroplasia: Current evidence and perspectives on the natural history

Julie Hoover-Fong, Moira S. Cheung, Virginia Fano, Lars Hagenas, Jacqueline T. Hecht, Penny Ireland, Melita Irving, Klaus Mohnike, Amaka C. Offiah, Ericka Okenfuss, Keiichi Ozono, Cathleen Raggio, Louise Tofts, Dominique Kelly, Renée Shediac, Wayne Pan, Ravi Savarirayan

• Publication

Achondroplasia Natural History Study (CLARITY): a multicenter retrospective cohort study of achondroplasia in the United States

Julie E. Hoover-Fong, Adekemi Y. Alade, S. Shahrukh Hashmi, Jacqueline T. Hecht, Janet M. Legare, Mary Ellen Little, Chengxin Liu, John McGready, Peggy Modaff, Richard M. Pauli, David F. Rodriguez-Buritica, Kerry J. Schulze, Maria Elena Serna, Cory J. Smid, Michael B. Bober

• Publication

Growth in achondroplasia including stature, weight, weight-for-height and head circumference from CLARITY: achondroplasia natural history study—a multi-center retrospective cohort study of achondroplasia in the US

Julie E. Hoover-Fong, Kerry J. Schulze, Adekemi Y. Alade, Michael B. Bober, Ethan Gough, S. Shahrukh Hashmi, Jacqueline T. Hecht, Janet M. Legare, Mary Ellen Little, Peggy Modaff, Richard M. Pauli, David F. Rodriguez-Buritica, Maria E. Serna, Cory Smid, Chengxin Liu, and John McGready

• Publication

Quality of life, physical functioning, and psychosocial function among patients with achondroplasia: a targeted literature review (tandfonline.com)

Constantinos Constantinides,Sarah H. LandisORCID Icon,James Jarrett,Jennifer Quinn, Penelope J. Ireland

• Publication

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

• Publication

Interindividual variability in transgene mRNA and protein production following adeno-associated virus gene therapy for Hemophilia A

Sylvia Fong, Bridget Yates, Choong-Ryoul Sihn, Aras N. Mattis, Nina Mitchell, Su Liu, Chris B. Russel, Benjamin Kim, Adebayo Lawal, Savita Rangarajan, Will Lester, Stuart Bunting, Glenn F. Pierce, K. John Pasi and Wing Yen Wong

• Publication

Real-World Rates of Bleeding, Factor VIII Use, and Quality of Life in Individuals with Severe Haemophilia A Receiving Prophylaxis in a Prospective, Noninterventional Study

Gili Kenet, Yeu-Chin Chen, Gillian Lowe, Charles Percy, Huyen Tran, Annette von Drygalski, Marc Trossaert, Mark Reding, Johannes Oldenburg, Maria Eva Mingot-Castellano, Young-Shil Park, Flora Peyvandi, Margareth C. Ozelo, Johnny Mahlangu, Jennifer Quinn, Mei Huang, Divya B. Reddy, Benjamin Kim

• Publication

Molecular analysis of AAV5-hFVIII-SQ vector-genome-processing kinetics in transduced mouse and nonhuman primate livers

Choong-Ryoul Sihn, Britta Handyside, Su Liu, Lening Zhang, Ryan Murphy, Bridget Yates, Lin Xie, Richard Torres, Chris B. Russel, Charles A. O’Neill, Erno Pungor, Stuart Bunting, Sylvia Fong.

• Publication

The Impact of Pre-existing Immunity on the Non-clinical Pharmacodynamics of AAV5-Based Gene Therapy

Brian R. Long, Krystal Sandza, Jennifer Holcomb, Lucy Crockett, Gregory M. Hayes, Jeremy Arena, Carls Fonck, Laurie S. Tsurda, Becky Schweighardt, Charles A. O’neill, Stephen Zoog, Christian Vettermann