Growth and Transformation - diagnosed 3-6 years

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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

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Han Phan, MD

Director

Rare Disease Research, LLC

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Jennifer Wallace Valdes, PT

CureDuchenne Certified Physical Therapist

CureDuchenne Physical Therapy Program

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Beginning the Journey - diagnosed 0-3 yearsGrowth and Transformation - diagnosed 3-6 yearsSpreading Wisdom - diagnosed 6+ years

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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Beginning the Journey - diagnosed 0-3 yearsGrowth and Transformation - diagnosed 3-6 yearsSpreading Wisdom - diagnosed 6+ years

2024 FUTURES Steroid Use

Breakout Session: A Steroid Discussion, One Disease, Many Paths

With more choices available, what should families consider? A panel breakout session to help families navigate the steroid journey, make informed choices, and proactively respond to side-effects.

Diana Castro, MD

Associate Professor of Pediatrics, Neurology and Neurotherapeutics

University of Texas Southwestern

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Nat Nasomyont

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Sheryl Marrazzo

Duchenne Mother, Grandmother and Advocate

4 Jake's Sake | CureDuchenne

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Patricia Brown

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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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Beginning the Journey - diagnosed 0-3 yearsGrowth and Transformation - diagnosed 3-6 yearsSpreading Wisdom - diagnosed 6+ years

Avidity Biosiences

Advancing Exon Skipping AOCs for the Treatment of Duchenne Muscular Dystrophy

Avidity Biosciences is a biotechnology company based in La Jolla, California. At Avidity, we are driven by our mission to improve the lives of people affected by diseases with limited therapeutic options, such as Duchenne muscular dystrophy (DMD). We are doing this by realizing the broad and disruptive potential of our Antibody Oligonucleotide Conjugates (AOC™) platform. AOCs combine the specificity of monoclonal antibodies and the precision of oligonucleotides. Avidity is advancing three different exon skipping AOC drugs designed to treat the root cause of DMD in individuals who are amenable to skipping exons 44, 45 or 51. Our first program in development for the treatment of DMD is called AOC 1044 and is designed to treat individuals who are amenable to exon 44 skipping treatments. We are planning to initiate our first clinical trial with AOC 1044 by the end of 2022.

Mark Stahl, MD, PhD

Senior Medical Director, Clinical Development

Avidity Biosciences

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Beginning the Journey - diagnosed 0-3 yearsGrowth and Transformation - diagnosed 3-6 yearsSpreading Wisdom - diagnosed 6+ years