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2024 FUTURES – Action is Therapy

Action is Therapy: Getting Involved With CureDuchenne Why I…fundraise, advocate, attend events, volunteer, engage with CureDuchenne. An overview of CureDuchenne Programs and the champions who are working to Cure Duchenne.

Paul and Laura Heaton

Calves to Cure DMD

Sheryl Marrazzo

Duchenne Mother, Grandmother and Advocate

4 Jake's Sake | CureDuchenne

2024 FUTURES – Clinical Trial Design, Lessons Learned and Implications for the Future

An analysis of combined clinical trial data and what it can teach about clinical trial design. What has gone right and what should be changed in the future?

Lauren Morgenroth, MS, CGC

Chief Operating Officer


Barry Byrne, MD, PhD

Professor and Associate Chair

University of Florida, Department of Pediatrics

Susan J Ward, PhD

Founder and Executive Director


2024 FUTURES – Therapies to Preserve and Protect Muscle

Presentations covering therapies aiming to preserve and protect muscle in Duchenne, including anti-inflammatory agents, muscle stabilizers, and other mechanisms with relevant updates from companies in the space.


Karyn Koladicz, MD

Executive Medical Director for Medical Affairs, Neurology PTC Therapeutics

Lianna Orlando, PhD

Senior Director of Research


Abby Bronson

Vice President, Patient Advocacy & External Innovation

Edgewise Therapeutics

Stanley Iyadurai, MD, PhD

Sr VP Medical Affairs and Drug Discovery


Scott Baver, PhD

Head of Medical Affairs

ITF Therapeutics

Mark Awadalla

Vice President of Clinical Operations


2024 FUTURES Exon skipping

A brief introduction on the rationale behind exon skipping therapies, followed by an overview of current and investigative exon skipping therapies.
Key insights were shared by Dr. Angel Angelov, Dr. Ash Dugar, Lianna Orlando, PhD, Dr. Kevin M. Flanigan, Laura Torrente, PhD, Alayna Tress, MPH, Karin Lucas, PhD, Mahasweta Girgenrath, Phd, Jessica Duis, MD, Husam Younis, PharmD, PhD.

Lianna Orlando, PhD

Senior Director of Research


Ash Dugar, PhD

Senior Vice President, Medical Affairs

Dyne Therapeutics

Kevin Flanigan, MD

Director, Center for Gene Therapy Nationwide Children's Hospital

Karin Lucas, Ph.D

West Medical Science Liaison Team Lead, Global Medical Affairs Sarepta Therapeutics

Laura Torrente, PhD

Alayna Tress, MPH

Mahasweta Girgenrath, Phd

Jessica Duis, MD

Husam Younis, PhD

Dr. Angel Angelov

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


Jahannaz Dastgir, DO

Clinical Development Lead REGENXBIO

Patrick Gonzalez, PhD

Senior Director of Clinical Science Solid Biosciences

Robyn van Poelgeest, MD, PhD


2024 FUTURES Keynote with Dr. Peter Marks, M.D., Ph.D

Dr. Peter Marks, M.D., Ph.D., director of the Center for Biologics Evaluation and Research (CBER) at the FDA, was our keynote speaker. CBER oversees the regulation and approval of all cell and gene therapies, including Sarepta’s Elevidys for 4-5-year-olds with Duchenne.

Dr. Peter Marks, M.D., Ph.D.

2024 FUTURES KEYNOTE, Embracing OUR Future

Adults with Duchenne as they explain how they have embraced their FUTURE with Duchenne. They will share their perspective on how they’ve navigated school, jobs, and relationships to live their most purposeful lives in the face of adversity.

DJ Kimble, Jake Marrazzo, Ryan Russell, PhD,
Hawken Miller


Hawken Miller

Content Strategist


Jake Marrazzo

Living with Duchenne

2024 FUTURES Multidisciplinary Proactive Care in Duchenne

Leading Duchenne providers discuss recommendations and advancements in their specialty and answer questions from the audience.

Dr. Aravindhan Veerapandiyan

Pediatric Neurologist

Arkansas Children's Hospital

Kent Williams, MD

Pediatric Gastroenterologist

Nationwide Children's Hospital

Barry Byrne, MD, PhD

Professor and Associate Chair

University of Florida, Department of Pediatrics