PPMD’s 2026 Annual Conference is in full swing!
PPMD’s 2026 Annual Conference kicked off with opening remarks from Katherine Beaverson, MD, CEO of PPMD, who welcomed attendees, introduced the dedicated PPMD team, recognized the many individuals and partners who strengthen our community, and shared how PPMD is working every day to accelerate research, improve care, and advocate for everyone living with Duchenne and Becker muscular dystrophy. Watch the introduction video here.
Research Highlights
On Day 1 of Conference, afternoon research sessions showcased promising advances across the Duchenne and Becker research landscape. Attendees heard updates on exon skipping therapies, followed by presentations on several PPMD-supported initiatives, including protein mapping, universal antibody screening, fat fraction modeling for clinical trials, and Tevard Biosciences’ nonsense variant tRNA gene therapy program. Together, these efforts reflect PPMD’s commitment to accelerating research and expanding therapeutic opportunities for the community.
ICYMI: Avidity Biosciences Submits Biologics License Application to FDA for del-zota
Dr. Paul Kaplan from Tevard Biosciences, who PPMD invested in through our Venture Pathways program, shared updates on the company’s development of a tRNA gene therapy that will address nonsense mutations. Also part of PPMD’s Gene Therapy Initiative, PPMD has provided funding to Dr. Michelle Rengarjan, who presented on her group’s efforts to develop universal antibody testing, addressing a major challenge for families today who experience challenges understanding all the different therapies they may or may not be eligible for.
Offering the community updates on projects supported by previous PPMD holiday fundraising campaigns, Dr. Alison Barnard presented updates from the Protein Mapping Project, which focused on 10-year proteomics data (biomarkers) to better understand disease progression. Dr. Sarah Kim presented on work, funded by PPMD through our Biomarkers Initiative, focused on refining models of fat fraction, captured by MRI, which allow for better prediction of an individual’s changes in function and can be utilized in drug development to get to answers faster.
Today’s Breaking News
Solid Biosciences Shares Updates on SGT-003
Kevin Flanigan, MD, from Nationwide Children’s Hospital and Principal Investigator in the Phase 1/2 INSPIRE DUCHENNE clinical trial, provided an overview of the SGT-003 development program at PPMD’s 2026 Annual Conference during the Gene Therapy: Today & Tomorrow session on Friday, June 26. This session is recorded and will be made available to the public as soon as possible within two weeks of the live event.
Capricor Therapeutics Shares Positive Five-Year HOPE-2 OLE Data for Deramiocel in Duchenne
Capricor will be presenting the five-year data from the HOPE-2 open label extension (OLE) study of Deramiocel at PPMD’s 2026 Annual Conference during a late breaking news session on Saturday, June 27, at 4:10 PM ET. This session will be recorded and made available to the public as soon as possible within two weeks of the live event.
Friday Keynote: Joel Wood
Joel Wood, President and Co-Founder of Federation to Eradicate Duchenne (FED), opened Day 2 of Annual Conference with a powerful keynote. Reflecting on his family’s Duchenne journey and his decades of patient advocacy, he shared lessons learned on the impact individuals can have when they channel their passion into meaningful action. Joel has been connected with PPMD since the organization’s early days, and has been a key partner in advocating for the needs of the Duchenne and Becker community before Congress. His message challenged attendees to recognize the unique role each person plays in advancing progress and reminded the community that lasting change begins when passion is transformed into purpose.
Topics in Care
Conversations around Duchenne and Becker care are always a highlight of PPMD’s Annual Conferences. So far this year, we’ve explored everything from genetics to managing inflammation, from navigating school to accessing approved therapies, and essential elements of care at every age and stage. We’ve also enjoyed sessions specific to Becker care, as well as a session dedicated to offering behind-the-scenes insights on PPMD care meetings and the work PPMD does with healthcare professionals to improve care for the community.
In addition to learning about cardiac care, PPMD’s session, Cardiac Research Therapies: Investigational and Repurposed Progress, focused on emerging therapies in development for dystrophinopathies. The discussion highlighted investigational approaches, including Deramiocel, ifetroban, and STM-01. This session was recorded and will be available to watch and listen to shortly after the conference concludes.
Kids & Sibs Tracks
No matter your age or your connection to Duchenne and Becker, the PPMD Annual Conference has something for everyone. This year, our Kids Track includes more than 80 children, offering opportunities to learn together, socialize, get creative, and—of course—have fun. These interactive sessions are thoughtfully designed by a team of educators who specialize in bringing children of all abilities together.
In addition to the Kids Track, PPMD’s Sibs Track is off and running, featuring social events, meetups, and dedicated time for siblings to connect and build community.
On Saturday, siblings will take the main stage for a candid panel, sharing their perspectives on growing up in a Duchenne household. Their voices will offer meaningful insight into the challenges, connections, and unique bonds that shape their journey. This session will be recorded and available for all to view after the PPMD Annual Conference concludes.
Teens & Young Adults
PPMD’s Adult Advisory Committee (PAAC) sessions for teens and adults have offered meaningful opportunities for connection, learning, and shared experiences throughout the conference. The PAAC kicked things off with a social gathering for the whole family on Wednesday night before diving into conversations on gaming as a tool for building social connections and relationships. Attendees also picked up practical advice during PAAC Hacks and Travel 101, where experienced community members shared tips to make traveling with Duchenne more manageable and enjoyable.
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