2024 FUTURES New Therapeutic Approaches to Address Unmet Needs

Presentations on early-stage research and development companies working on approaches to address the unmet therapeutic needs in the Duchenne community.

Sharif Tabebordbar, PhD

Lianna Orlando, PhD

Courtney Young, PhD

2024 FUTURES Preparing for the Unexpected, Responding to Emergencies

A Duchenne emergency case study, the steps to take, and the signs to watch for. From implementing an emergency plan and following it to effective communication with the Emergency Room to get the care you need. A review of the cautions and a proactive plan of care from specialists.

Mindy Cameron

Patient Advocacy Lead

Santhera Pharmaceuticals

Han Phan, MD

Director

Rare Disease Research, LLC

Jennifer Wallace Valdes, PT

CureDuchenne Certified Physical Therapist

CureDuchenne Physical Therapy Program

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

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

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

CureDuchenne

Jake Marrazzo

Living with Duchenne

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

CureDuchenne

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

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 – 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

TRiNDS

Barry Byrne, MD, PhD

Professor and Associate Chair

University of Florida, Department of Pediatrics

Susan J Ward, PhD

Founder and Executive Director

cTAP