Spreading Wisdom - diagnosed 6+ years

Clear all

Involucrarse con CureDuchenne

Una cálida bienvenida del equipo de CureDuchenne y unas palabras de algunas familias campeonas de CureDuchenne sobre cómo lograron el éxito en la recaudación de fondos al unir a su comunidad.

Paul and Laura Heaton

Calves to Cure DMD

Tim and Laura Revell

CureDuchenne Champion Family

Linking the Community to a Cure

CureDuchenne Link is a centralized data hub which combines clinical data, biosamples, and patient reported data for individuals with Duchenne and Becker muscular dystrophy, as well as carriers. Information and samples provided by participants are coded and available for use by approved researchers across the world.

Ana Christensen, MPH

Lead Project Manager, Strategic Solutions

TRiNDS

Más allá de la distrofina

Esta sesión de la conferencia brinda información sobre las terapias emergentes más allá de la restauración de la distrofina, así como actualizaciones/anuncios de las empresas en este espacio.

Han Phan, MD

Director

Rare Disease Research, LLC

Mindy Cameron

Patient Advocacy Lead

Santhera Pharmaceuticals

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

Chief Operating Officer

PTC Therapeutics

Abby Bronson

Vice President, Patient Advocacy & External Innovation

Edgewise Therapeutics

Daniel Paulson, MD

Vice President of Clinical Development

Capricor Therapeutics

Next Generation Technology Panel

A brief overview of the rationale behind next generation technology, as well as presentations from the companies in this space. Moderated discussion included.

Erin Brubaker

Chief Operating Officer

Code Bio

Michael Kelly, PhD

Chief Scientific Advisor

CureDuchenne

Joe McIntosh, MD

Chief Medical Officer

Gennao Bio

Genine Winslow, MSc

CEO & President

Chameleon Biosciences