1 2 aim



Implementing the updated standards of care that are informed by best practices by experts in the field.


Sharing research interests, exploring research opportunities, co-funding, sharing knowledge, avoiding duplication.


Encourage and inform people living with Duchenne and organizations to use their voice to inform policy in their country.


Enhance the relationship with regulatory and HTA & extend influence on policy makers.


Engage and educate people living with Duchenne and organizations about diagnosis, standards of care, research, drug development and access to therapies.


Advocating to lessen the burden in clinical trials, expand inclusion criteria, thoughtful consideration of invasive procedures.

Clinical Trials

Including the patient voice in clinical trials by expanding inclusion criteria, lessen burden of participation, innovative designs, minimal exposure to placebo and fair pricing policies


Invest in data & knowledge capturing, promoting open science, data interoperability and security in accordance with the FAIR principles.

Outcome measures

Support the need for sensitive outcomes measures, validated biomarkers, and harmonization of regulatory decisions and working with all stakeholders in delivering access to therapies.