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RARE Scholars Program Provides Support to College Students Living with Genetic Conditions

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Where Are They Now? Catching Up with Alumni of Our RARE Scholars Program

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RARE Scholars Program

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Howard University Partners with BioMarin on Regulatory Fellowship

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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

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Community Commitment

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New Data to be Presented for BioMarin’s VOXZOGO® (vosoritide) in Children with Hypochondroplasia and Achondroplasia at the American College of Medical Genetics and Genomics (ACMG) Annual Clinical Genetics Meeting

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Dennis J. Slamon

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Global Compliance and Ethics Program

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Experiences of children and adolescents living with achondroplasia and their caregivers

Renée Shediac, Olga Moshkovich, Heather Gerould, Rachel Ballinger, Agnes Williams, M. Alex Bellenger, Jennifer Quinn, Julie Hoover-Fong, Klaus Mohnike, Ravi Savarirayan, Dominique Kelly

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Persostence of haemostatic response following gene therapy with valoctocogene roxaparvovec in severe hemophilia A

K John Pasi, Michael Laffan, Savita Rangarajan, Tara M Robinson, Nina Mitchell, Will Lester, Emily Symington, Bella Madan, Xinqun Yang, Benjamin Kim, Glenn F Pierce, Wing Yen Wong

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Assessing the potential cost-effectiveness of a gene therapy for the treatment of hemophilia A

Keziah Cook, Shaun P. Forbes, Kelly Adamski, Janice J. Ma, Anita Chawla & Louis P. Garrison Jr

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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

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Efficacy and safety of enzyme replacement therapy with BMN 110 (elosulfase alfa) for Morquio A syndrome (mucopolysaccharidosis IVA): a phase 3 randomised placebo-controlled study

Christian J. Hendriksz, Barbara Burton, Thomas R. Fleming, Paul Harmatz, Derralynn Hughes, Simon A. Jones, Shuan-Pei Lin, Eugen Mengel, Maurizio Scarpa, Vassili Valayannopoulos, Roberto Giugliani, STRIVE Investigators, Peter Slasor, Debra Lounsbury, Wolfgang Dummer