Strength in Community:Engaging the Community

Strength in Community:Engaging the Community


As a company co-founded by those touched by Duchenne muscular dystrophy, we aim to be a center of excellence for advancing the science of Duchenne and other rare neuromuscular and cardiac diseases. Our deep commitment to the patient community is at the heart of what we do. For every approach we take and decisions we make, we put the needs of patients and their families at the forefront.

We ensure that the patient voice and perspective is at the core of the company’s DNA, woven into every step of our research, development, and clinical programs. We value our relationships with the patient community – from advocacy organizations to individual patients and families – and continually engage in discussions to help ensure our scientific and clinical pursuits address the unmet needs of patients, their caregivers, and families with a sense of urgency.

Our dedication to discover and develop novel therapeutic approaches for rare neuromuscular and cardiac diseases guides us every day. We are motivated by the prospect that there will be an end to these devastating diseases and remain committed to doing our part to advance the rigorous science that combats them.

Please feel free to contact us with questions and ideas:

Annie Ganot
VP, Head of Patient Advocacy