Just a few years ago, families attending CureDuchenne’s FUTURES 2026 Annual Conference were weighing whether to participate in clinical trials. Today, the conversation has shifted dramatically. With multiple approved therapies now available for Duchenne muscular dystrophy (DMD), families and clinicians face a new question: how should these treatments be used together?
During a FUTURES panel discussion moderated by CureDuchenne Chief Medical Advisor Brenda Wong, MD, experts Aravindhan Veerapandiyan, MD, Diana Castro, MD, and Stephen Chrzanowski MDhttps://providers.clevelandclinic.org/provider/szymon-krzyzanowski/4269143, explored the evolving landscape of combination therapies with gene therapy, exon skippers, , emerging treatments and standard of care steroid treatment. The session covered the providers’ approach/strategy of combination therapy for their patients, discussed the different treatment needs of patients in different stages of DMD, the clinical monitoriing for outcomes and safety of combined treatments and the impact of the use of combined treatments on clinical trial participation and design. Families participated actively in the question-and-answer session.
More Treatment Options, More Complex Decisions
The panel emphasized that current therapies are not cures. Instead, they are designed to slow disease progression, maintain mobility and independence, and improve quality of life.
Dr. Aravindhan Veerapandiyan noted that “None of them is a cure. They’re not stopping your disease progression, and they’re not reversing the disease process. It’s more of slowing of disease progression,” while speaking on the different treatment options.
Dr. Veerapandiyan described today’s treatments as falling into two broad categories:
- Therapies that target dystrophin production, such as gene therapy and exon skipping
- Therapies that target downstream effects of the disease, including inflammation and muscle degeneration
As more treatments become available, clinicians are increasingly considering how these approaches may work together rather than as standalone options.
There Is No One-Size-Fits-All Approach
One of the strongest messages from the session was that treatment decisions must be individualized.
While families often ask which therapy is best, the panelists explained that there is no universal answer. Factors such as age, mutation type, disease stage, treatment goals, access to care, transportation challenges, and family circumstances all influence decision-making.
Dr. Castro noted that even practical considerations matter. Some families may struggle to travel for frequent monitoring appointments or laboratory testing, while others may prioritize minimizing treatment burden to maintain quality of life. These realities must be part of every treatment conversation.
The panel encouraged families to ask questions, understand potential benefits and risks, and work closely with their care teams to develop a plan that aligns with their goals and circumstances.
Steroids Remain an Important Foundation
Despite excitement surrounding new therapies, the panelists stressed that corticosteroids remain a cornerstone of Duchenne care.
Inflammation begins early in Duchenne and contributes to ongoing muscle damage. Steroids continue to play a critical role in slowing that process.
At the same time, clinicians acknowledged the significant side effects associated with long-term steroid use, including bone health complications, weight gain, and other challenges. As new therapies emerge, researchers hope to better understand whether new treatments would provide some steroid sparing effect for some individuals to be able to reduce steroid exposure.
For now, however, steroids remain an important standard of care for many individuals living with Duchenne.
Why Timing Matters
The panel repeatedly returned to one important concept: time matters.
“Duchenne is progressing, period,” Dr. Castro emphasized, explaining that muscle damage begins long before symptoms become obvious. Waiting for a future therapy may mean losing valuable time and muscle function that cannot be recovered later.
While families naturally want the best available treatment, the experts encouraged thoughtful consideration of currently available options rather than delaying care in hopes that a future therapy may arrive.
As newborn screening expands and earlier diagnosis becomes possible, these decisions may become even more important.
Measuring Success Beyond the Clinic
A recurring question throughout the session was how clinicians can determine whether combination therapies are working.
Traditional measures such as the North Star Ambulatory Assessment and timed function tests remain valuable, but the panel acknowledged their limitations. Improvements that matter to families do not always translate into measurable changes on standardized assessments.
Some examples shared during the discussion included:
- Improved endurance during daily activities
- Better hand function
- Increased independence
- reduced caregiving demands
- Greater stability and mobility
The panel also discussed emerging tools such as MRI imaging, digital monitoring devices, gait analysis, wearable technologies, and artificial intelligence-driven assessments that may help capture meaningful changes in the future.
The Challenge of Access
While scientific progress continues, access remains a significant concern.
Many therapies require extensive monitoring, laboratory testing, and coordination between specialists. Families living far from major Duchenne centers may face additional barriers, including travel costs, missed work, and challenges accessing specialized care.
The panel emphasized the importance of strong partnerships between specialty centers, primary care providers, and local healthcare teams. Building a “medical home” model can help ensure families receive necessary monitoring while reducing travel burdens whenever possible.
The discussion also highlighted broader challenges related to treatment costs and insurance coverage, particularly as combination therapies become more common.
Looking Ahead
Despite the complexity of today’s treatment landscape, the panel closed on a hopeful note.
Just a decade ago, families had few disease-modifying options. Today, clinicians and families are discussing gene therapy, exon skipping, anti-inflammatory treatments, and emerging combination approaches. While many questions remain about how best to sequence and combine therapies, the progress has been remarkable.
As Dr. Chrzanowski reflected, the fact that clinicians and families are now debating among multiple treatment options is itself evidence of how far the Duchenne community has come.
Key Takeaways for Families
- Combination therapy is becoming an important part of Duchenne care for individuals with Duchenne.
- Treatment decisions should be individualized based on each person’s needs and goals.
- Steroids remain an important standard of care for many patients.
- Earlier intervention may help preserve function over time.
- Quality of life should remain central to treatment decisions.
- Access, monitoring requirements, and family circumstances are critical factors to consider.
- The treatment landscape for Duchenne is evolving rapidly, creating new opportunities and new questions for families and clinicians alike.
While there is still much to learn, one thing is certain: the Duchenne community has entered a new era of treatment, and families now have more options—and more hope—than ever before.
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The CureDuchenne Cares team provides personalized guidance, educational resources, and one-on-one support to help families make informed care decisions throughout every stage of Duchenne.
- Register for the CureDuchenne FUTURES Annual Conference to hear the latest in Duchenne care
This content is intended for educational and informational purposes only and should not be considered medical advice. Always consult your healthcare provider before making decisions about your medical care or treatment plan.
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