Beginning the Journey - diagnosed 0-3 years

Clear all

2021 FUTURES: Keynote Panel

An inspiring conversation about living in the moment with a rare diagnosis.

Sean Baumstark

Co-Founder

Two Disabled Dudes

Kyle Bryant

Co-Founder

Two Disabled Dudes

Jake Marrazzo

Living with Duchenne

Glenn Olesen

Living with Duchenne

GlennDane Adapt and Conquer

Tanner Rico

Living with Duchenne

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

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

Ana Christensen, MPH

Lead Project Manager, Strategic Solutions

TRiNDS

Leslie Porter

Family, Duchenne Advocate

Family, Duchenne Advocate

Michael Kelly, PhD

Chief Scientific Advisor

CureDuchenne

Michaela Walker, MPH

Project Manager

University of Kansas Medical Center

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

Beyond Dystrophin Panel

This conference session provides insight and updates from companies whose therapies target critical aspects of the Duchenne disease process that occur alongside reductions in dystrophin.

Abby Bronson

Vice President, Patient Advocacy & External Innovation

Edgewise Therapeutics

Mindy Cameron

Patient Advocacy Lead

Santhera Pharmaceuticals

Matthew Klein, M.D., M.S., FACS

Chief Operating Officer

PTC Therapeutics

Daniel Paulson, MD

Vice President of Clinical Development

Capricor Therapeutics

Han Phan, MD

Director

Rare Disease Research, LLC