• Board Profile

Elaine J. Heron

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Robert J. Hombach

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Richard A. Meier

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Maykin Ho

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V. Bryan Lawlis

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David E.I. Pyott

• News Post

Raising a Child with Hemophilia: BioMarin Employee Jess Klass Shares Her Story

• 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

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

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*

• Publication

Valoctogene Roxaparvovec Gene Therapy for Hemophilia A

Margareth C. Ozelo, M.D., Ph.D., Johnny Mahlangu, M.B., B.Ch., M.Med., K. John Pasi, M.D., Ph.D., Adam Giermasz, M.D., Ph.D., Andrew D. Leavitt, M.D., Michael Laffan, M.D., Ph.D., Emily Symington, M.D., Doris V. Quon, M.D., Ph.D., Jiaan-Der Wang, M.D., Ph.D., Kathelijne Peerlinck, M.D., Ph.D., Steven W. Pipe, M.D., Bella Madan, M.D., et al., for the GENEr8-1 Trial Group*

• 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

• Publication

Gene Therapy with BMN 270 Results in Therapeutic Levels of FVIII in Mice and Primates and Normalization of Bleeding in Hemophilic Mice

Stuart Bunting, Lening Zhang,1 Lin Xie, Sherry Bullens, Rajeev Mahimkar, Sylvia Fong, Krystal Sandza, Danielle Harmon, Bridget Yates, Britta Handyside, Choong-Ryoul Sihn, Nicole Galicia, Laurie Tsuruda, Charles A. O’Neill, Anil Bagri, Peter Colosi, Shinong Long, Gordon Vehar, and Barrie Carte

• Publication

Validation of an IFN-gamma ELISpot assay to measure cellular immune responses against viral antigens in non-human primates

Fan Yang, Kathryn Patton, Theresa Kasprzyk, Brian Long, Soumi Gupta, Stephen J Zoog, Kristin Tracy, Chrstian Vettermann