August 1- 4, 2025 • Philadelphia, PA
Team Titin, together with our partners Cure CMD and A Foundation Building Strength for Nemaline Myopathy, proudly hosted the 2025 SciFam Conference – a four-day gathering for the congenital muscular dystrophy, nemaline myopathy, and titinopathy communities.
More than 344 attendees – including affected individuals, families, caregivers, clinicians, researchers, and industry representatives – came together to learn, discover, share stories, and build lasting connections. Families had the rare opportunity to sit shoulder-to-shoulder with world-class doctors and scientists, sharing their lived experiences firsthand. Meanwhile, affected individuals found community and support by connecting with peers who truly understand their journey.
Conference Highlights
- 60 Sessions Across 4 Days: Covering care, research, advocacy, quality of life, and community connections.
- 3 Titinopathy Breakout Sessions: Focusing on genetics & diagnostics, cardiac care, and research updates.
- 100 Researchers & Clinicians: Sharing the latest science, trial readiness strategies, and innovations in care.
- 9 Industry Representatives: From pharma and health industries, highlighting strong collaborations.
- Community Programming: Caregiver wellness, advocacy training, social events, and more – all designed to foster connection beyond research and care.
Kids Camp: From Magic to Mayhem
For children attending SciFam, the Kids Camp offered a world of fun – from a science show and wildlife encounter to a magician/juggler, face painting, tattoos, and even a dance party. It wasn’t just about the activities; it was about joy, friendship, and belonging. Kids Camp created unforgettable memories for the children while giving parents and caregivers peace of mind to fully participate in sessions.
Young Adult Programming
Teens and young adults had their own space to connect, learn, and share openly. Sessions included icebreakers, peer meetups, and practical workshops on finances, independence, and inclusion. Clinicians joined to answer questions about healthcare now and into the future. Open conversations around intimacy, identity, and relationships created safe spaces for supportive dialogue.
Scientific Meeting: Advancing Research Together
The final two days of SciFam were dedicated to the Scientific Symposium, where more than 100 leading neuromuscular researchers and clinicians gathered to share progress and challenges. Key themes included:
- Latest Advances in Therapeutics: Gene therapy updates, novel treatment strategies, and disease modeling.
- Biomarkers & Natural History Studies: Essential tools to track disease progression and prepare for clinical trials.
- Shared Approaches Across Subtypes: While genetic origins differ, many therapies under study may benefit multiple conditions, accelerating progress across the neuromuscular field.
Relive the Experience
Couldn’t make it or want to revisit your favorite moments?
- Session recordings are being added to the 2025 SciFam Team Titin Playlist on YouTube in the coming weeks.
- Explore 800+ photos on our CrowdPic page, and if you snapped your own pictures, we’d love for you to add them!
Gratitude
SciFam 2025 was only possible thanks to the dedication of our attendees, families, volunteers, organizers, and sponsors. Together, we created something unforgettable – and together, we are stronger.
“Attending the SciFam 2025 conference was such a meaningful experience for me and our family. Meeting other families who understand our journey reminded me that we’re not alone. Hearing from and speaking directly with specialists gave us valuable insights and practical knowledge to better support our son. I left feeling more informed, connected, and hopeful about the road ahead.”
– a 2025 SciFam attendee
