Brett Crawford, PhD

Title: Executive Director and Head of the Musculoskeletal Therapeutic Area

Organization: BioMarin Pharmaceutical Inc.

Bio: Brett E. Crawford, PhD is Executive Director and Head of the Musculoskeletal Therapeutic Area at BioMarin Pharmaceutical. He has over 20 years of experience in preclinical development of therapeutics for rare genetic diseases. Dr. Crawford founded Zacharon Pharmaceuticals in 2004 where he led the scientific strategy focused on glycan-targeted therapeutics for rare genetic diseases. At BioMarin since 2014, the Musculoskeletal Therapeutic Area has brought multiple programs to the clinic including the recently approved Voxzogo for Achondroplasia. Brett received his BS in Biological Sciences with honors from UC Davis and his PhD in Biomedical Sciences from UC San Diego.

Exon Skipping and Read Through Agents Panel

A brief introduction on the rationale behind mechanisms of endogenous dystrophin restoration, like exon skipping and read through agent therapies, followed by critical information and updates from the companies in this space. Moderated discussion included.

Barry Byrne, MD, PhD

Professor and Associate Chair

University of Florida, Department of Pediatrics

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Jay Chauhan, PharmD

Medical Science Liaison

NS Pharma

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Brett Crawford, PhD

Executive Director and Head of the Musculoskeletal Therapeutic Area

BioMarin Pharmaceutical Inc.

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Ash Dugar, PhD

Senior Vice President, Medical Affairs

Dyne Therapeutics

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Xiao Shelley Hu, PhD, MS

VP

Wave Life Sciences

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Matthew Klein, M.D., M.S., FACS

Chief Operating Officer

PTC Therapeutics

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Nerissa Kreher, MD, MBA

Chief Medical Officer

Entrada Therapeutics

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James McArthur, PhD

President and Chief Executive Officer

PepGen

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Mark Stahl, MD, PhD

Senior Medical Director, Clinical Development

Avidity Biosciences

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Deanna Tucker, PharmD

Senior Medical Science Liaison

Sarepta Therapeutics

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