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MPS IVA

• News Post

A 100-Mile Road to Diagnosis: A Mother Shares Her Son’s Journey with Hypochondroplasia

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Global Medical Information Form (2)

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Global Compliance and Ethics Program

• Publication

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

• Publication

Systematic Review and Meta-Analysis of Neuropsychiatric Symptoms and Executive Functioning in Adults With Phenylketonuria

Deborah A. Bilder, J. Kay Noel, Erin R. Baker, William Irish, Yinpu Chen, Markus J. Merilainen, Suyash Prasad, and Barbara J. Winslow

• News Post

Caring for Children with Achondroplasia: An Interview with Dr. Ciara McDonnell

• News Post

BioMarin Teams Use Genetic Expertise and Computational Tools to Target More Common Conditions

• News Post

BioMarin Pioneer Honored through New Building Name and Book Launch

• News Post

At the Intersection of Science and Art

• News Post

The Unforgettable Moments from ‘The Style Show: A Runway to Empower’

• News Post

The Three Lessons I’ve Learned from Planning a Fashion Show That Are Applicable to Life

• News Post

It’s Time to Give a Platform, or in My Case “Runway,” to People with Rare Disease

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Community Commitment

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PKU

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BioMarin Community Guidelines

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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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RARE Scholars 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