For Every Life Touched by Rare Disease
Our mission is to bring innovative treatments to those who need them most, through care, collaboration, and compassion.
Patient Focus
How We’re Making a Difference
We are deeply committed to actively partnering and continuously collaborating with patient advocacy groups, individuals, and families throughout every stage of their journey.
Research & Innovation
Pushing the boundaries of science to develop life-changing treatments.
Patient Care & Support
Enhancing patient well-being through dedicated care and resources.
Community & Advocacy
Championing awareness, support, and policy advancements for impact.
Our Patient-Centric Mission
Our core mission is to significantly enhance the quality of life for patients and their families. We are leading a “crusade” driven by the belief that no one should be left behind by their disease.
Engaging the Community
We are deeply committed to fostering strong relationships with our customers – physicians and clinicians – as well as with patients and caregivers. Our strategy focuses on education, support, and collaboration.
Patient & Family Engagement
We prioritize community engagement throughout the drug development process, offering educational programs, accessible resources, webinars, and interactive platforms to foster understanding and support. Personal stories and lived experiences help inform our work and guide our communications.
Advocacy Group Partnerships
We maintain continuous engagement with a broad network of support and advocacy organizations to align our development efforts with community needs and priorities.
Physician/Clinician Focus
Physicians and clinicians are essential scientific partners in our development strategy. We tailor our communications to align with their clinical expertise and evidence-based expectations.
Therapeutic Benefits
For patients and their physicians, our therapies are designed to stabilize muscle tissue, promote regeneration, and serve as a complementary approach to treatments such as gene therapy. We are committed to developing a patient-friendly therapy and building a comprehensive support network to enhance long-term care and outcomes.
