The journey to find treatments for rare congenital muscle disease, Nemaline Myopathy, is a collective effort, and A Foundation Building Strength (AFBS) has been at the forefront of this mission. Recently, the organization concluded a groundbreaking 10-part webinar series titled “Amplifying the NM Community’s Voice,” made possible by the Patient-Centered Outcomes Research Institute (PCORI). This series focused on advancing patient-centered research outcomes, highlighting the importance of community involvement in the quest to find treatments for Nemaline Myopathy (NM).
Webinar Recap
The final webinar in this series served as a culmination of insights and discussions that unfolded throughout the project. AFBS values patient participation and aimed to build a network that encourages engagement in research development for NM. The agenda covered a variety of crucial topics, including a recap of key learnings from the project, a review of AFBS’ mission and vision, and discussions about patient opportunities and next steps.
Key Learnings and Mission
Throughout the series, NM world experts and researchers generously shared their expertise with the NM community. The webinars delved into research fundamentals, the importance of natural history studies, and the significance of genetic confirmation for precision treatments. AFBS emphasized the role of translational research in expediting treatment development and highlighted ongoing projects funded by the foundation.
Engaging the Community
The series acknowledged the diverse NM community, dedicating specific sessions to different cohorts. The adult-affected community, in particular, played a vital role in guiding the strategic plan development. AFBS recognized the importance of making connections, being vulnerable, and understanding where the foundation could grow to better serve the community.
Community Challenges and Voices Heard
A survey involving 117 NM community members, identified the greatest challenges faced by individuals and caregivers, further guiding research investments. There was also emphasis on ways for the community to actively participate in research, including joining the Congenital Muscle Disease International Registry (CMDIR), obtaining genetic confirmation, and contributing tissue samples to the NM BioBank at Beggs Lab (Boston Children’s Hospital).
New Resources and Initiatives
AFBS introduced valuable resources such as the NM Resource Kit, the NM Awareness Card, the new AFBS website, all designed to spread awareness, better understand NM and build the NM community. The foundation also highlighted community gatherings like SciFam and regional gatherings, fostering connections and shared experiences among NM families.
Lessons Learned and Community Feedback
AFBS shared lessons learned throughout the journey, emphasizing the importance of community engagement and responsibility. The foundation recognized the day-to-day challenges faced by those managing health with NM and expressed a commitment to supporting research for effective treatments. Community feedback reinforced the significance of small contributions to impact research positively.
Looking to the Future
As AFBS looks to the future, the organization is extending invitations for community members to join the newly forming Community Advisory Team. This team will provide input on mission-based activities, further integrating the voices of those affected by NM into the foundation’s strategic initiatives.
The AFBS webinar series has been a transformative experience, bringing together experts, researchers, and the NM community to advance patient-centered research outcomes. With strategic priorities focused on therapy development, financial stability, organizational growth, and community engagement, AFBS is poised to make monumental progress in the mission to find treatments for Nemaline Myopathy.
Next Steps
- Provide feedback via post-webinar survey
- Join the Registry (CMDIR.org)
- Complete the Contact List survey
- Join Nemaline and Congenital Myopathy Community group on Facebook
- Follow the Nemaline Myopathies page (Facebook)
- Follow A Foundation Building Strength for Nemaline Myopathy on Facebook, Instagram and YouTube