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

Tiffany Cook, MS, CCC-SLP

Senior Director

CureDuchenne Cares

Michael Kelly, PhD

Chief Scientific Advisor

CureDuchenne

Sheryl Marrazzo

Duchenne Mother, Grandmother and Advocate

4 Jake's Sake | CureDuchenne

Christina Trout, RN, MSN

Advanced Practice Nurse, Neuromuscular Program

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

Jennifer Wallace Valdes, PT

CureDuchenne Certified Physical Therapist

CureDuchenne Physical Therapy Program

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

Olivier Danos, PhD

Chief Scientific Officer

REGENXBIO

Emil Kakkis, PhD

CEO & President

Ultragenyx Pharmaceutical

Michael Kelly, PhD

Chief Scientific Advisor

CureDuchenne

Alison Mcvie-Wylie, PhD

Vice President, Disease Area Executive, DMD

Vertex Pharmaceutical

Carl Morris, PhD

Chief Scientific Officer

Solid Biosciences

Eric Olson, PhD

Chief Scientific Advisor

Vertex Pharmaceuticals

Matthew Pletcher, PhD

Division Head of Gene Therapy Research & Technical Operations

Astellas Gene Therapies

Deanna Tucker, PharmD

Senior Medical Science Liaison

Sarepta Therapeutics

Genine Winslow, MSc

CEO & President

Chameleon Biosciences

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

Emil Kakkis, PhD

CEO & President

Ultragenyx Pharmaceutical

Alison Mcvie-Wylie, PhD

Vice President, Disease Area Executive, DMD

Vertex Pharmaceutical

Dan Levy, MD

VP and Development Head, Rare Neurology Pfizer

Carsten Bönnemann, MD

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

Kevin Flanigan, MD

Director, Center for Gene Therapy Nationwide Children's Hospital

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

Jahannaz Dastgir, DO

Clinical Development Lead REGENXBIO

Patrick Gonzalez, PhD

Senior Director of Clinical Science Solid Biosciences

Robyn van Poelgeest, MD, PhD

Sarepta

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

Barry Byrne, MD, PhD

Professor and Associate Chair

University of Florida, Department of Pediatrics

Carsten Bönnemann, MD

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

Kevin Flanigan, MD

Director, Center for Gene Therapy Nationwide Children's Hospital

Robyn van Poelgeest, MD, PhD

Sarepta

Sharif Tabebordbar, PhD

Co-Founder and Chief Scientific Officer

Kate Therapeutics

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

Ana Christensen, MPH

Lead Project Manager, Strategic Solutions

TRiNDS

Leslie Porter

Family, Duchenne Advocate

Family, Duchenne Advocate

Michael Kelly, PhD

Chief Scientific Advisor

CureDuchenne

Michaela Walker, MPH

Project Manager

University of Kansas Medical Center