Project Summary
A Foundation for Building Strength for Nemaline Myopathy will engage stakeholders—including patients, caregivers, researchers, physicians, health payers, other advocacy organizations, policy makers and biotechnology innovation centers—through targeted meetings, webinars, and an annual conference and knowledge exchange. The collaboration with stakeholder groups is expected to develop a clear path to treatment for the rare disease, foster better patient-physician communication, improve patient care, and further encourage patient-centered research.
The projected outputs from this project are six teleconferences, five webinars, five in-person meetings and an annual conference.
Project collaborators include The Hospital for Sick Children; NINDS/NIH; Boston Children's Hospital; MIT NEW DIGS; NORD; University of Florida Muscle Lab; multi-stakeholder volunteers; multi-stakeholder meeting panelists; and conference consultants.