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2024 FUTURES GENE Therapy panel

Gene Therapy Approaches to Treatment We covered the rationale behind gene therapy approaches for Duchenne and received updates from Pfizer, Sarepta Therapeutics, REGENXBIO Inc., and Solid BioSciences.

Michael Kelly, PhD

Chief Scientific Advisor

CureDuchenne

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Jahannaz Dastgir, DO

Clinical Development Lead REGENXBIO

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Patrick Gonzalez, PhD

Senior Director of Clinical Science Solid Biosciences

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Robyn van Poelgeest, MD, PhD

Sarepta

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Beginning the Journey - diagnosed 0-3 yearsGrowth and Transformation - diagnosed 3-6 yearsSpreading Wisdom - diagnosed 6+ years

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