Resource Library Archive - Page 6 of 10

2021 FUTURES: Gene Therapy & Gene Editing Symposium

Information and updates on the gene therapy and gene editing landscape from experts in the field.

**We experienced a technical issue during this session. Please reach out with questions to Futures@CureDuchenne.org if needed.

Beth Belluscio, MD, PhD

Global Clinical Lead, Rare Neurological Disorders

Pfizer

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Olivier Danos, PhD

Chief Scientific Officer

REGENXBIO

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Emil Kakkis, PhD

CEO & President

Ultragenyx Pharmaceutical

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Michael Kelly, PhD

Chief Scientific Advisor

CureDuchenne

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Alison Mcvie-Wylie, PhD

Vice President, Disease Area Executive, DMD

Vertex Pharmaceutical

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Carl Morris, PhD

Chief Scientific Officer

Solid Biosciences

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Eric Olson, PhD

Chief Scientific Advisor

Vertex Pharmaceuticals

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Matthew Pletcher, PhD

Division Head of Gene Therapy Research & Technical Operations

Astellas Gene Therapies

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Deanna Tucker, PharmD

Senior Medical Science Liaison

Sarepta Therapeutics

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Genine Winslow, MSc

CEO & President

Chameleon Biosciences

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CureDuchenne Cares Educational Webinar: Reimagining Quality of Life in Duchenne

This webinar, in partnership with the University of Kansas Medical Center, explores topics like achieving milestones, quality of life, and setting goals in families navigating Duchenne muscular dystrophy. The panelists also discuss how to incorporate quality of life metrics into clinical care.

Jeffrey M. Statland, MD

Professor of Neurology

University of Kansas Medical Center

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Kelli Reiling Ott

Clinical Assistant Professor

University of Kansas Medical Center

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Vincent Czerwinski, MD

Medical Student

University of Kansas Medical Center

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Tiffany Cook, MS, CCC-SLP

Senior Director

CureDuchenne Cares

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Doug Levine, PT

CureDuchenne Certified Physical Therapist

CureDuchenne

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Navigating Clinical Trials

This resource offers a view of Clinical Trials 101: what they are, how they run, and how to make decisions surrounding participating in a clinical trial.