In 2025, Team Titin continued to grow, connect, and create change for people living with, caring for, and researching titin (TTN)–related muscle and heart conditions. Guided by our four mission pillars—Connection, Advocacy, Care, and Research—we made meaningful progress together. None of this would be possible without our families, donors, partners, clinicians, and researchers who believe in this mission as deeply as we do.
Here’s a look at what we achieved in 2025 – and what we’re excited to build in 2026.
A Landmark Year for Connection
One of our proudest accomplishments in 2025 was co-hosting the SciFam Conference, our largest and most ambitious event to date. Over four powerful days, we brought together families, clinicians, researchers, advocates, and industry partners to learn, share, and connect. Recorded sessions from our conference can be viewed on our YouTube channel in the “Team Titin SciFam 2025 Videos” playlist.
SciFam 2025 Highlights:
- 60 sessions across care, research, advocacy, and quality of life
- 3 titin-focused breakout sessions on genetics, cardiac care, and research updates
- Over 100 clinicians and researchers sharing the latest science
- 9 industry partners strengthening collaboration
- Caregiver wellness sessions, advocacy training, and community-building events
This wasn’t just a conference – it was a gathering of a growing, hopeful community.
Our community also grew in powerful ways throughout the year:
- 168 individuals in our CMDIR registry
- Over 500 affected individuals and families receive our newsletter
- More than 200 clinicians and scientists connected through Team Titin
- Social media growth across Facebook, Instagram, YouTube, and our website, reaching thousands of new people
Every new connection strengthens our shared voice and our shared future.
Advocacy: Amplifying the Titin Community Voice
In 2025, we continued to advocate for recognition, resources, and representation for the titin community.
Our advocacy work included:
- Participating in the MDA Neuromuscular Advocacy Collaborative
- Hosting an advocacy session at SciFam where community members shared their lived experiences
- Continuing work toward an ICD code for titinopathy (pending publication of a consensus paper on nomenclature)
- Attended the 2025 International Limb-Girdle Muscular Dystrophy Conference
We are committed to ensuring that titin families are seen, heard, and represented in healthcare systems and research priorities.
Supporting Care for Today
While we push for treatments of tomorrow, we also focus on helping families get the best care today.
In 2025, we:
- Maintained and updated our care resources online
- Shared care guides directly with newly connected families
- Contributed as patient representative to an international publication on congenital myopathies
- Partnered in heart health and rare disease awareness campaigns throughout the year
- Took part in global cardiomyopathy and neuromuscular advocacy efforts
We also began work with Muscular Dystrophy UK (MDUK) to create a TTN factsheet that is still in draft with the goal of bringing trusted information to even more families worldwide.
Advancing Research and Hope
Research is at the heart of Team Titin’s mission and 2025 was a year of real momentum.
We supported research by:
- Co-hosting international case conferences and science update with experts with record attendance
- Supporting the CMDIR, our patient registry
- Partnering with Critical Path Institute (C-Path) to accelerate treatments as part of the LGMDs Task Force
- Sharing de-identified data to help advance researcher
- Chaired the session, “Rare Recessive LGMD Subtypes” at the LGMD Conference including titin expert Dr. Bjarne Udd
Research Funding in 2025:
- $50,000 grant to Genethon in France for TTN muscle organoid research
- Received updates on research funded with the Silvey Family Foundation in 2024 to Jennifer Roggenbuck
- $25,000 in additional funding for the TREATIN Natural History Study in collaboration with ZNM Zusammen Stark!
These projects are helping build the foundation for future clinical trials and treatments.
At SciFam’s Scientific Symposium, over 100 experts gathered to share progress on gene therapy, biomarkers, and natural history studies – showing just how much progress is happening across the neuromuscular field.
Looking Ahead: Our Priorities for 2026
We are entering 2026 with hope, purpose, and clear priorities.
Connection
- Hosting a heart health webinar in January 2026, “Heart-Healthy Lifestyle Tools for People with Titin (TTN) – Related Heart Conditions”
- Expand outreach through Google Ad Grants
- Deepen relationships with affected families and MDUK
Care
- Finalize and publish the TTN factsheet with MDUK
- Launch Team Titin on the Citizen Health platform so families can:
- Organize medical records in one place
- Simplify care coordination
- Use AI-powered tools to understand their health
- Advance research through data in a way that respects registrants privacy preferences
Advocacy
- Continue work toward an ICD code for titinopathy
- Participate in national neuromuscular advocacy meetings
Research
- Continue and expand grant funding
- Support ongoing organoid and natural history studies
- Share information about clinical trial opportunities
- Participate in major scientific meetings in 2026, including chairing a session on titinopathy at the MDA Clinical & Scientific conference
- We are also proud to partner with Citizen Health to help families contribute de-identified data – accelerating research that could change lives.
Thank You for Being Part of This
Everything we achieved in 2025 happened because of you – families who shared their stories, donors who believed in this mission, researchers and clinicians who partnered with us, and volunteers who gave their time and hearts.
We are proud of what we’ve built together. And we are even more excited about what’s ahead.
Here’s to a future filled with stronger connections, better care, louder advocacy, and real progress toward treatments for titin-related conditions.
Together, we are Team Titin. And together, we strengthen our community.