Spreading Wisdom - diagnosed 6+ years

Clear all

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

See more from same speaker

Han Phan, MD

Director

Rare Disease Research, LLC

See more from same speaker

Jennifer Wallace Valdes, PT

CureDuchenne Certified Physical Therapist

CureDuchenne Physical Therapy Program

See more from same speaker

Video
Share this:
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

See more from same speaker

Barry Byrne, MD, PhD

Professor and Associate Chair

University of Florida, Department of Pediatrics

See more from same speaker

Carsten Bönnemann, MD

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

See more from same speaker

Kevin Flanigan, MD

Director, Center for Gene Therapy Nationwide Children's Hospital

See more from same speaker

Robyn van Poelgeest, MD, PhD

Sarepta

See more from same speaker

Sharif Tabebordbar, PhD

Co-Founder and Chief Scientific Officer

Kate Therapeutics

See more from same speaker

Video
Share this:
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

See more from same speaker

Nat Nasomyont

See more from same speaker

Sheryl Marrazzo

Duchenne Mother, Grandmother and Advocate

4 Jake's Sake | CureDuchenne

See more from same speaker

Patricia Brown

See more from same speaker

Video
Share this:
Beginning the Journey - diagnosed 0-3 yearsGrowth and Transformation - diagnosed 3-6 yearsSpreading Wisdom - diagnosed 6+ 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

See more from same speaker

Ana Christensen, MPH

Lead Project Manager, Strategic Solutions

TRiNDS

See more from same speaker

Leslie Porter

Family, Duchenne Advocate

Family, Duchenne Advocate

See more from same speaker

Michael Kelly, PhD

Chief Scientific Advisor

CureDuchenne

See more from same speaker

Michaela Walker, MPH

Project Manager

University of Kansas Medical Center

See more from same speaker

Video
Share this:
Beginning the Journey - diagnosed 0-3 yearsGrowth and Transformation - diagnosed 3-6 yearsSpreading Wisdom - diagnosed 6+ years

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

See more from same speaker

Diana Castro, MD

Founder and Director of the Neurology and Neuromuscular Care Center

See more from same speaker

Husam Younis, PhD

See more from same speaker

Sarah Boyce

President and CEO of Avidity Biosciences

See more from same speaker

Video
Share this:
Beginning the Journey - diagnosed 0-3 yearsSpreading Wisdom - diagnosed 6+ years