Investigational Duchenne Muscular Dystrophy Treatments to Watch in 2026
Duchenne muscular dystrophy (DMD) is a rare, inherited neuromuscular condition caused by mutations in the dystrophin gene, which plays a critical role in protecting muscle cells during everyday movement. Without enough functional dystrophin, muscles are more prone to damage and gradually weaken over time. DMD primarily affects boys and usually begins in early childhood, with early signs often including difficulty keeping up physically with peers. As the condition progresses, muscle weakness becomes more widespread and can eventually affect the arms, heart, and breathing muscles, making it a lifelong condition that requires ongoing care. While advances in supportive treatment have improved quality of life and life expectancy, there is currently no cure for DMD. Most existing therapies focus on slowing disease progression, preserving muscle function for as long as possible, and managing complications rather than correcting the underlying genetic cause. Treatment is further complicated by the wide range of genetic mutations involved, meaning that some therapies are only applicable to certain individuals, and even promising approaches may raise questions about long-term effectiveness and safety.
This article highlights Duchenne muscular dystrophy clinical trials to watch in 2026, focusing on studies that are:
- recently registered or ongoing
- testing experimental treatments
- listed on public research registries
Important: This article is for informational purposes only. Being listed here does not mean a therapy works or is recommended. Clinical trial participation should always be discussed with a healthcare professional.
Below are five Duchenne muscular dystrophy clinical trials involving experimental treatments that represent different research approaches.
1. Elevidys (Delandistrogene Moxeparvovec) Gene Therapy Study
What is this study evaluating?
This study is evaluating whether Elevidys can deliver a micro-dystrophin gene into skeletal muscle cells using a single intravenous infusion. Because the full dystrophin gene cannot fit inside commonly used viral vectors, Elevidys uses a shortened version that retains structural regions needed to anchor muscle fibers during contraction. The study is examining whether muscle cells can produce this micro-dystrophin after treatment and whether expression persists beyond the initial months following delivery.
How could this approach change the way Duchenne is treated?
Most Duchenne therapies do not alter the structural weakness of muscle fibers caused by missing dystrophin. Elevidys directly targets that weakness by introducing dystrophin production inside muscle cells rather than reducing damage after it occurs. The key question this study addresses is whether a single gene delivery can produce dystrophin at levels that remain detectable over time, which would distinguish gene therapy from treatments that require repeated dosing or lifelong administration.
Study link:
https://clinicaltrials.gov/study/NCT05096221
2. DYNE-251 (DELIVER) Exon-Skipping Therapy Trial
What is this study evaluating?
This study is evaluating DYNE-251, an exon-skipping therapy designed for people with Duchenne muscular dystrophy who have a specific type of genetic change called an exon 51–amenable mutation. In Duchenne, some mutations interrupt the dystrophin gene so badly that muscle cells cannot make any dystrophin at all. Exon skipping works by teaching the cell to skip over the damaged section of the gene, allowing it to make a shorter version of dystrophin instead of none.
DYNE-251 is built differently from earlier exon-skipping drugs. It uses a delivery system designed to help more of the therapy actually reach muscle cells, which has been a major limitation of previous exon-skipping treatments. This study is examining whether this improved delivery allows muscle cells to consistently produce dystrophin rather than only small, scattered amounts.
How could this approach change the way Duchenne is treated?
Earlier exon-skipping therapies showed that dystrophin production was possible, but the amount made in muscle tissue was often very low and uneven. This made it unclear whether the protein produced was enough to meaningfully affect muscle stability. This study is focused on whether better delivery can overcome that limitation and lead to more widespread dystrophin production within muscle.
The central question being addressed is whether exon skipping can move beyond a limited molecular effect and produce a biological change that is sustained across muscle tissue. Answering that question is necessary to determine whether exon-skipping therapies can play a larger role in Duchenne treatment rather than remaining highly constrained by delivery challenges.
Study link:
https://clinicaltrials.gov/study/NCT05524883
3. PF-06939926 Gene Therapy Study
What is this study evaluating?
This study is evaluating PF-06939926, an experimental gene therapy that uses an adeno-associated virus (AAV) to deliver dystrophin-related genetic instructions into muscle cells. In Duchenne muscular dystrophy, muscle cells lack the instructions needed to make dystrophin, which leaves them structurally weak and prone to damage. This therapy is designed to deliver genetic material that helps muscle cells produce a dystrophin-associated protein inside the cell.
Because gene therapies rely on viral carriers to deliver genetic instructions, this study is closely focused on how well the therapy spreads through the body and reaches muscle tissue after a single intravenous dose. It is also examining whether muscle cells can begin producing dystrophin-related protein following delivery and whether that production persists over time.
How could this approach change the way Duchenne is treated?
Gene therapy approaches differ in how efficiently they deliver genetic material to muscle and how the body responds to that delivery. Earlier gene therapies have faced challenges related to immune response, delivery consistency, and durability of gene expression. This study is specifically addressing whether this AAV-based approach can deliver dystrophin-related genetic material in a way that muscle cells can use consistently rather than temporarily.
The central question being explored is whether this delivery method can support sustained dystrophin-related protein production inside muscle cells without triggering responses that limit how long the therapy remains effective. Answering this question is critical for determining whether gene therapies like PF-06939926 can move from short-term biological activity toward more durable treatment strategies for Duchenne.
Study link:
https://clinicaltrials.gov/study/NCT04281485
4. RGX-202 (AFFINITY DUCHENNE) Gene Therapy Study
What is this study evaluating?
This study is evaluating RGX-202, an investigational gene therapy designed to deliver a functional dystrophin gene into muscle cells using a next-generation viral delivery system. In Duchenne muscular dystrophy, muscle cells lack dystrophin, which normally helps muscle fibers maintain their structure during contraction. RGX-202 is designed to provide muscle cells with genetic instructions to produce dystrophin internally.
Unlike earlier gene therapies that use standard delivery vectors, RGX-202 uses a different vector design intended to improve how efficiently the gene reaches muscle tissue. This study is examining whether this delivery method allows dystrophin expression within muscle cells after treatment and how muscle tissue responds following gene delivery.
How could this approach change the way Duchenne is treated?
A major challenge in Duchenne gene therapy has been delivering enough genetic material to muscle tissue without provoking immune responses that limit effectiveness. Earlier gene therapies have sometimes struggled with uneven delivery across muscles or limited durability of gene expression. This study is addressing whether a modified delivery system can improve how dystrophin genes are distributed within muscle tissue and how long that expression remains detectable.
The key question being explored is whether changes in vector design can overcome delivery-related limitations seen in prior gene therapy efforts. Answering that question is necessary to determine whether refinements in gene delivery technology can lead to more consistent and scalable gene-based treatment strategies for Duchenne.
Study link:
https://clinicaltrials.gov/study/NCT05693142
5. Long-Term Follow-Up of Duchenne Gene Therapy Recipients
What is this study evaluating?
This study is evaluating the long-term effects of investigational gene therapies that were previously given to individuals with Duchenne muscular dystrophy. Unlike trials that test a new treatment, this study follows people who already received gene therapy to observe what happens years after the initial dose.
The study is examining whether dystrophin-related protein production remains detectable over time, how muscle tissue behaves long after treatment, and whether any delayed safety issues emerge as participants are followed over extended periods. Because gene therapies are typically given once, understanding what happens long after delivery is a central focus of this research.
How could this approach change the way Duchenne is treated?
Most gene therapy trials focus on short-term outcomes measured months after treatment. This study addresses a different and unresolved question: whether the biological effects of gene therapy persist or fade over time, and how the body responds in the long run.
The findings from this study help clarify whether gene therapies for Duchenne provide sustained biological activity or whether their effects diminish, which directly affects how these treatments are monitored and evaluated. Understanding long-term durability and safety is essential for determining how gene therapies fit into Duchenne care beyond the initial treatment period.
Study link:
https://clinicaltrials.gov/study/NCT05689164
Final Takeaway
These Duchenne muscular dystrophy clinical trials to watch in 2026 reflect the wide range of research strategies currently being explored — from gene therapy and exon-skipping approaches to long-term follow-up studies.
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Disclaimer
This article is for informational purposes only. The information shared here summarizes publicly available research, clinical trials, and scientific developments and no guarantees are made regarding accuracy, completeness, or current relevance. Always consult a qualified healthcare professional regarding any health-related decisions.