Beginning the Journey - diagnosed 0-3 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 Family Guide to Dealing With Duchenne Muscular Dystrophy

This resource presents the history of Duchenne muscular dystrophy and provides a basic understanding of the genetics, management and treatment of the diesease.

Samiya Ahmad, MD

Assistant Professor of Pediatrics, Child Neurology and Sleep Medicine | Medical Director, Comprehensive Neuromuscular Center

Baylor College of Medicine | Children's Hospital of San Antonio

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Beginning the Journey - diagnosed 0-3 years

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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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 Duchenne Journey 

From FUTURES 2023

 

  • Coping as a Caregiver
  • Communicating Duchenne to your child/family
  • ​Preparing for a bright future: play, communicate, and appropriate exercise
  • Neurological concerns
  • Introducing Stretching and Equipment
  • Role of Social Work
  • Duchenne’s Impact on Nutritional Health and Habits

Kayla Richards, LMSW

Social Worker

UT Health Austin Pediatric Neurosciences at Dell Children's

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Doug Levine, PT

CureDuchenne Certified Physical Therapist

CureDuchenne

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Leslie Porter

Family, Duchenne Advocate

Family, Duchenne Advocate

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Diana Castro, MD

Associate Professor of Pediatrics, Neurology and Neurotherapeutics

University of Texas Southwestern

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Kindann Fawcett, PhD

Post Doctoral Fellow

University of Arkansas for Medical Sciences

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