Michael Kelly, PhD

Title: Chief Scientific Advisor

Organization: CureDuchenne

Bio: Michael Kelly, PhD is a senior pharmaceutical executive and brings more than 30 years of experience in drug discovery and development to the organization. As CureDuchenne’s Chief Scientific Advisor, he is responsible for advancing drug development programs and identifying new drug targets that exhibit potential to transform the treatment of Duchenne muscular dystrophy.

2021 FUTURES Quality of Life Panel: Making Decisions

Focused on empowering families to make informed decisions about their child’s care / personal care across all junctures of the Duchenne journey.

John Brandsema, MD

Child Neurologist and Neuromuscular Section Head

Children’s Hospital of Philadelphia

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Tiffany Cook, MS, CCC-SLP

Senior Director

CureDuchenne Cares

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Michael Kelly, PhD

Chief Scientific Advisor

CureDuchenne

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Sheryl Marrazzo

Duchenne Mother, Grandmother and Advocate

4 Jake's Sake | CureDuchenne

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Christina Trout, RN, MSN

Advanced Practice Nurse, Neuromuscular Program

Department of Pediatrics, University of Iowa Stead Family Children’s Hospital

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Jennifer Wallace Valdes, PT

CureDuchenne Certified Physical Therapist

CureDuchenne Physical Therapy Program

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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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2023 FUTURES: Friday Keynote – On the Cutting Edge of Research

The science and research-focused day of FUTURES will begin with an engaging keynote panel featuring key opinion leaders who will discuss the successes, learnings, challenges, and future of this disease space.

Michael Kelly, PhD

Chief Scientific Advisor

CureDuchenne

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Emil Kakkis, PhD

CEO & President

Ultragenyx Pharmaceutical

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Alison Mcvie-Wylie, PhD

Vice President, Disease Area Executive, DMD

Vertex Pharmaceutical

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Dan Levy, MD

VP and Development Head, Rare Neurology Pfizer

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Carsten Bönnemann, MD

Chief, Neuromuscular and Neurogenetic Disorders of Childhood Section National Institute of Neurological Disorders and Stroke/NIH

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Kevin Flanigan, MD

Director, Center for Gene Therapy Nationwide Children's Hospital

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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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2024 FUTURES Responding to AAV Based Treatment Challenges

A moderated panel discussion focusing on the potential challenges and complications of AAV therapy and the clinical strategies to make dosing AAV more successful.

Michael Kelly, PhD

Chief Scientific Advisor

CureDuchenne

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Barry Byrne, MD, PhD

Professor and Associate Chair

University of Florida, Department of Pediatrics

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Carsten Bönnemann, MD

Chief, Neuromuscular and Neurogenetic Disorders of Childhood Section National Institute of Neurological Disorders and Stroke/NIH

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Kevin Flanigan, MD

Director, Center for Gene Therapy Nationwide Children's Hospital

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Robyn van Poelgeest, MD, PhD

Sarepta

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Sharif Tabebordbar, PhD

Co-Founder and Chief Scientific Officer

Kate Therapeutics

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A Holistic View of Clinical Trials

This panel discussion provides a robust conversation about how clinical trials intersect with family life in the Duchenne community.

Tiffany Cook, MS, CCC-SLP

Senior Director

CureDuchenne Cares

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Ana Christensen, MPH

Lead Project Manager, Strategic Solutions

TRiNDS

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Leslie Porter

Family, Duchenne Advocate

Family, Duchenne Advocate

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Michael Kelly, PhD

Chief Scientific Advisor

CureDuchenne

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Michaela Walker, MPH

Project Manager

University of Kansas Medical Center

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Avidity Biosciences: Presentation and discussion on initial data from EXPLORE44 Clinical Trial

This webinar, hosted by CureDuchenne and Avidity Biosciences, presents the initial data from the EXPLORE 44 clinical trial. The trial evaluates the efficacy and safety of Dalzota (AOC 1044) for treating Duchenne muscular dystrophy (DMD) in patients amenable to exon 44 skipping. Key findings include a significant increase in exon skipping and dystrophin production, with a notable reduction in creatine kinase levels, indicating potential muscle health improvements. The discussion also covers the future plans for the trial and the urgency to advance treatment options for DMD patients.

Michael Kelly, PhD

Chief Scientific Advisor

CureDuchenne

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Diana Castro, MD

Founder and Director of the Neurology and Neuromuscular Care Center

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Husam Younis, PhD

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Sarah Boyce

President and CEO of Avidity Biosciences

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CureDuchenne Webinar with Wave Life Sciences

Wave will provide an overview of initial clinical trial results for WVE-N531 in individuals with Duchenne muscular dystrophy who are amenable to exon 53 skipping, as well as an overview of the potentially registrational FORWARD-53 study. 

Anne-Marie Li-Kwai-Cheung, MChem, MTOPRA, RAPS

Chief Development Officer

Wave Life Sciences

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Michael Kelly, PhD

Chief Scientific Advisor

CureDuchenne

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