It meets bi-annually in Amsterdam (or virtually) after initiating a call of interest. Following the call of interest, an invitation is extended to the company for an individualized session. In addition, 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.
Consulting with the Duchenne CAB provides certain benefits:
- Attaining first-hand knowledge of living with DMD;
- Gaining insights into patient needs and preferences;
- Receiving unbiased advice in order to design optimal and meaningful clinical trials that better fit patient needs, thereby improving recruitment, adherence and retention.