PROJECT OVERVIEW

Duchenne Community Advisory Board (CAB)

The Duchenne CAB is an independent, autonomous and international board of trained patient representatives (parents) from 12 different countries, currently: US, Canada, UK, Netherlands, Belgium, Germany, France, Spain, Italy, Romania, Turkey and Israel.

It meets bi-annually in Amsterdam (or virtually) after initiating a call of interest, following which an invitation is extended to the company for an individualized session. Upon prior agreement, ad hoc advice is available based on specific urgent questions or issues.

The goal of the Duchenne CAB is to provide its collective knowledge and expertise in a common endeavor to accelerate research, development, clinical trials and access to effective treatments for DMD worldwide.

Some benefits of consulting with the Duchenne CAB are: to attain first-hand knowledge of living with DMD; gain insights into patient needs and preferences; receive unbiased advice in order to design optimal and meaningful clinical trials that better fit patient needs, thereby improving recruitment, adherence and retention.

The core elements of the Duchenne CAB are absolute confidentiality to promote:

  • TRUST between all participants and provide a safe harbor for meaningful interaction
  • TRANSPARENCY to guarantee impartiality and integrity to enable maximization of the value and impact of insights and advice given to our industry partners: to promote an
  • EXCHANGE of ideas, knowledge, best practices and data sharing to accelerate drug development in DMD, optimize research and development and ensure optimal, meaningful and successful trials
  • ACTION PLAN to streamline cooperation between stakeholders, de-risk development and decrease cost from basic research to clinical trials, market approval, and access and reimbursement in a global environment.

Reports of the Duchenne CAB meetings can be found here:

For questions and inquiries please contact the Duchenne CAB Coordinator, Sally Hofmeister: [email protected]