The much-anticipated 2025 SciFam Conference, held from August 1-5 in Philadelphia, has concluded, and AFBS finds itself newly re-energized by the connections made and progress shared! This vital gathering brought together individuals and families affected by NM, Congenital Muscular Dystrophy (CMD), and Titinopathy, alongside a passionate group of clinicians and researchers from around the globe. We were proud to partner with Cure CMD and Team Titin to make this powerful event a reality, marking a significant step toward future Nemaline Myopathy research breakthroughs.
SciFam 2025 served as a crucial hub where science and community met, fostering a spirit of collaboration and shared purpose. The conference was designed to provide valuable information and support for families, while creating an essential feedback loop for the scientific and medical communities. This invaluable direct engagement ensures that research is guided by the real-world needs and experiences of those living with these rare neuromuscular diseases.
Affected families and individuals left Philadelphia having forged new friendships and a network of support, as well as a sense of excitement after hearing updates on AFBS-funded research projects focused on developing effective treatments for Nemaline Myopathy!
Check Out These Key Sessions!
We are thrilled to announce the release of the first four videos of the conference’s educational sessions, including two key plenary sessions and two NM focused sessions available now for viewing. These sessions offer a glimpse into the depth and breadth of the content shared at SciFam.
Orthopedics & Physical Therapy: Navigating NM Care
This plenary session brought together a panel of leading experts to discuss critical orthopedic and physical therapy topics. The panelists provided invaluable guidance on preparing for spine correction surgery, prioritizing different therapies, and balancing an active lifestyle with the need to prevent overuse. They answered questions on topics including oral motor therapy, scoliosis, and early interventions for infants, providing a comprehensive overview of how to build a stronger understanding of neuromuscular care.
Nutrition & Gastroenterology for Neuromuscular Disease
In this essential session, a panel of experts offered compassionate and practical guidance on navigating feeding challenges and gastrointestinal issues. They discussed critical topics like making the difficult decision to use a feeding tube, navigating growth charts for children with lower muscle mass, and the benefits of using blenderized foods and commercially available food blends. The discussion also included managing common bowel issues and understanding the risks and benefits of various supplements.
NM Community: Connect & Take Action
This session was a powerful reminder of our community’s strength. Attendees shared their own tips and tricks for navigating life with Nemaline Myopathy, which created a valuable repository of shared knowledge. The discussion highlighted a number of clever hacks for daily care, from ventilator tubing modifications to do-it-yourself supplement solutions. We also introduced new AFBS initiatives designed to empower the community. This included the Perspectives and Priorities Survey, a vital tool for collecting anonymous data that will be used to guide AFBS’s strategic initiatives and provide valuable insights to researchers and clinicians. The NM Awareness Advocacy Honors was also announced as a new annual program to celebrate individuals and families who have gone above and beyond to raise awareness and fundraise. Leticia Machado shared creative and successful fundraising ideas from her own community, illustrating that anyone can make a significant contribution to funding groundbreaking research through personal efforts like hosting a dinner party or organizing a local event. These initiatives give everyone a chance to play an active role in building a brighter future for the NM community.
Nemaline Myopathy Research Breakthroughs & Updates
The research session was filled with exciting news from our AFBS-funded scientists. This session provided updates on the latest progress on research projects, including promising early results on gene therapies for ACTA1 and KBTBD13-based Nemaline Myopathy and the repurposing of small molecule drugs with broad therapeutic potential. Attendees also heard about exciting new research on soft robotic garments designed to assist with movement. They learned how they can contribute to a new retrospective digital natural history study in partnership with Citizen Health as well as participate in our upcoming Perspective Natural History Study, part of a global collaboration. These breakthroughs represent significant momentum for the Nemaline Myopathy research field.
More to Come!
These videos are just a sampling of the many valuable educational sessions we’ll be releasing over the next few weeks. Be sure to stay tuned for even more content from SciFam!
SciFam 2025 was a tremendous success, and we extend our most sincere gratitude to every family who traveled to share their experiences and every researcher and clinician who dedicated their time and expertise. This collective effort is what drives us as we continue our mission to find treatments for Nemaline Myopathy, and we look forward to seeing the fruits of this collaboration in the years to come!
A Foundation Building Strength (AFBS) is a nonprofit organization dedicated to accelerating the development of treatments for Nemaline Myopathy. Our mission is to fund cutting-edge research to find effective treatments while providing resources and a strong community for families affected by NM. We are proud to be at the forefront of driving Nemaline Myopathy research breakthroughs forward through collaborative events like SciFam.
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