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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

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

Doug Levine, PT

CureDuchenne Certified Physical Therapist

CureDuchenne

Leslie Porter

Family, Duchenne Advocate

Family, Duchenne Advocate

Diana Castro, MD

Associate Professor of Pediatrics, Neurology and Neurotherapeutics

University of Texas Southwestern

Kindann Fawcett, PhD

Post Doctoral Fellow

University of Arkansas for Medical Sciences

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

Community of Caregivers

An interactive discussion on the unique journey of caregivers, and tools to unite and strengthen the Duchenne community in their shared experience.

Laura McLinn

Founder

Best Day Ever Foundation

Marissa Penrod

Co-Founder

The Duchenne Family Assistance Program

Leslie Porter

Family, Duchenne Advocate

Family, Duchenne Advocate

Comunidad de cuidadores

Una discusión interactiva sobre el viaje único de los cuidadores y herramientas para unir y fortalecer a la comunidad de Duchenne en su experiencia compartida.

Laura McLinn

Founder

Best Day Ever Foundation

Marissa Penrod

Co-Founder

The Duchenne Family Assistance Program

Leslie Porter

Family, Duchenne Advocate

Family, Duchenne Advocate

Conectando familias con la investigacion

El enfoque del seminario web de hoy es ayudar a educar y conectar a las familias con el panorama de los ensayos clínicos.

Amaris Sánchez-Larragoity, PsyD

Psicóloga licenciada

Diana Castro, MD

Associate Professor of Pediatrics, Neurology and Neurotherapeutics

University of Texas Southwestern