Vision

Our vision is to pioneer a future where every individual affected by Limb Girdle Muscular Dystrophy Type 2I/R9 can be treated early and live a life unencumbered by its challenges. By relentlessly pursuing innovative research, collaborative partnerships, and drug development, we envision accessible and effective treatments that slow or prevent the progression of the disease.

Goals

1. 1.Increase advocacy efforts and engage the LGMD community to participate in storytelling and reaching out to local, state and federal legislators to encourage support of rare disease initiatives.
2. 2.Educate our patient community on standards of care, natural history studies, patient registries, conferences and clinical trial opportunities. Identify patient advocates/ambassadors in each of the 50 states to share burden of rare disease stories and participate in legislative activities.
3. 3.Expanding number of advisory board members, volunteers and staff to expand our programs and offerings to the LGMD patient community. Added a community engagement coordinator to help onboard new volunteers and connect to the greater community for support.