Condition

Duchenne Muscular Dystrophy clinical trials and research

• 24Clinical trials
• 50NIH research projects
• 26Countries

Newest Duchenne Muscular Dystrophy clinical trials

All recruiting trials (24)

• VM100: a nutritional supplement to see if it improves quality of life for boys with Duchenne

  This Phase 2 study tests VM100, a nutritional drink, in boys aged 8 and up with Duchenne muscular dystrophy to see if it can improve quality of life and reduce fatigue.

  Recruiting Phase 2 University of Florida · Gainesville, Florida · NCT07766980

• KER-065 for Duchenne Muscular Dystrophy: safety and potential benefit in boys and men

  This study tests whether KER-065 is safe and can help with symptoms in males with Duchenne muscular dystrophy, including both ambulatory and nonambulatory participants who are on standard care.

  Recruiting at 2 sites Phase 2 Keros Therapeutics, Inc. · Tbilisi and 3 other locations · NCT07704099

• GNR-097 gene therapy for Duchenne muscular dystrophy in boys

  This study tests a gene therapy (GNR-097) to see if it is safe, tolerable, and potentially beneficial for ambulatory boys with Duchenne muscular dystrophy.

  Recruiting at 6 sites Phase 1/2 Ao Generium · Minsk and 5 other locations · NCT07673809

• ELEVIDYS safety in Duchenne patients treated after market approval

  This study tests the safety of ELEVIDYS, with sirolimus as a protective treatment, in male children and young adults with Duchenne Muscular Dystrophy who have previously received ELEVIDYS or are eligible to receive it commercially.

  Recruiting Phase 4 Sarepta Therapeutics, Inc. · Houston, Texas · NCT07542314

• SGT-003 gene therapy for ambulant boys and young men with Duchenne muscular dystrophy (IMPACT DUCHENNE)

  This trial will test whether a single intravenous infusion of SGT-003 can help ambulatory boys and young men with Duchenne muscular dystrophy maintain or improve walking and motor function.

  Recruiting at 4 sites Phase 3 Solid Biosciences Inc. · Little Rock, Arkansas and 6 other locations · NCT07160634

• Evaluating Givinostat for Young Children with Duchenne Muscular Dystrophy

  This study is testing a new drug called Givinostat to see if it's safe and how it works in young children aged 2 to 6 with Duchenne Muscular Dystrophy.

  Recruiting at 7 sites Phase 2 Italfarmaco · Brussels and 8 other locations · NCT06769633

• Evaluating respiratory functions and exercise capacity in children with neuromuscular diseases

  This study is trying to see how well children with Duchenne Muscular Dystrophy and Spinal Muscular Atrophy can breathe and exercise compared to healthy kids.

  Recruiting Lokman Hekim University · Ankara, Çankaya · NCT06756633

• Cell-based therapy for Duchenne Muscular Dystrophy

  This study is testing a new cell-based treatment for people with Duchenne muscular dystrophy to see if it is safe and helps improve muscle function.

  Recruiting Phase 1 Masonic Cancer Center, University of Minnesota · Minneapolis, Minnesota · NCT06692426

• Observing long-term safety of Vamorolone in boys with Duchenne Muscular Dystrophy

  This study is looking at how safe the drug Vamorolone is for boys with Duchenne Muscular Dystrophy and how it affects their quality of life over five years.

  Recruiting at 8 sites Catalyst Pharmaceuticals, Inc. · Phoenix, Arizona and 32 other locations · NCT06564974

• Gamified occupational therapy for adolescents with Duchenne muscular dystrophy

  This study is testing a fun, game-based therapy to see if it helps teenagers with Duchenne muscular dystrophy feel better and do everyday activities more easily compared to regular therapy.

  Recruiting Lokman Hekim University · Ankara · NCT06402942

• Combining exercise and tadalafil for treating Duchenne muscular dystrophy

  This study is testing if combining exercise with a medication called tadalafil can help boys with Duchenne muscular dystrophy improve their muscle function and blood flow.

  Recruiting Phase 2 University of Florida · Gainesville, Florida · NCT06290713

• Gene therapy for Duchenne muscular dystrophy

  This study is testing a new gene therapy called SGT-003 to see if it is safe and effective for children with Duchenne muscular dystrophy.

  Recruiting at 15 sites Phase 1/2 Solid Biosciences Inc. · Little Rock, Arkansas and 14 other locations · NCT06138639

• Using wearable technology to assess blood sugar and heart health in boys with Duchenne muscular dystrophy

  This study is testing how blood sugar levels and heart health are connected in boys with Duchenne muscular dystrophy using wearable technology to gather data.

  Recruiting Vanderbilt University Medical Center · Nashville, Tennessee · NCT06124196

• Using wearable technology to monitor blood sugar and heart health in boys with Duchenne Muscular Dystrophy

  This study is testing how wearable technology can help monitor blood sugar and heart health in boys with Duchenne Muscular Dystrophy to better understand their risks for heart problems.

  Recruiting Vanderbilt University Medical Center · Nashville, Tennessee · NCT06093100

• Collecting data on muscle disorders using wearable devices

  This study is testing a wearable device to track movement in people with muscle disorders like Duchenne Muscular Dystrophy and Myotonic Dystrophy to see how it can help improve their care.

  Recruiting at 2 sites Centre Hospitalier Universitaire de Liege · Liège and 7 other locations · NCT05982119

• Evaluating the effects of givinostat in boys with Duchenne muscular dystrophy who cannot walk

  This study is testing if a new drug called givinostat can help boys with Duchenne muscular dystrophy who can't walk by slowing down muscle decline over 18 months.

  Recruiting at 19 sites Phase 3 Italfarmaco · Leuven and 28 other locations · NCT05933057

• Lower dose corticosteroid treatment for infants with Duchenne Muscular Dystrophy

  This study is testing if a lower dose of corticosteroids can help infants and young boys with Duchenne Muscular Dystrophy improve their movement skills while causing fewer side effects than a higher dose.

  Recruiting at 4 sites Phase 4 Nationwide Children's Hospital · Chicago, Illinois and 3 other locations · NCT05412394

• Developing new biomarkers for muscular dystrophies

  This study is trying to find easier and less painful ways to measure how serious muscular dystrophies are by looking at urine and blood samples and using simple tests, to help improve diagnosis and treatment for people with these conditions.

  Recruiting at 4 sites Massachusetts General Hospital · Boston, Massachusetts and 4 other locations · NCT05019625

• Delandistrogene moxeparvovec gene therapy for Duchenne muscular dystrophy

  This trial tests a one-time gene therapy called delandistrogene moxeparvovec in children and young adults with Duchenne muscular dystrophy to try to restore dystrophin production and improve muscle function.

  Recruiting at 11 sites Phase 1 Sarepta Therapeutics, Inc. · Little Rock, Arkansas and 11 other locations · NCT04626674

• Observational study on Duchenne Muscular Dystrophy

  This study looks at how Duchenne Muscular Dystrophy and similar conditions develop over time in patients to help improve their care.

  Recruiting First Affiliated Hospital of Fujian Medical University · Fuzhou · NCT04012671

• Natural history of Duchenne muscular dystrophy in boys aged 4–9

  This project will follow boys aged 4–9 with Duchenne muscular dystrophy for 6–36 months to collect standard clinical, imaging, and lab data and create a baseline before possible gene therapy.

  Recruiting at 12 sites Genethon · Brussels and 14 other locations · NCT03882827

• Long-term Givinostat treatment for safety and tolerability in Duchenne muscular dystrophy

  This long-term treatment will try Givinostat in people with Duchenne muscular dystrophy who previously took the drug to see if it remains safe and tolerable.

  Recruiting at 3 sites Phase 2/3 Italfarmaco · Sacramento, California and 38 other locations · NCT03373968

• An online registry for Duchenne and Becker muscular dystrophy patients

  This study is creating an online registry to help people with Duchenne and Becker muscular dystrophy connect with clinical trials and find helpful information about their condition.

  Recruiting The Duchenne Registry · Washington D.C., District of Columbia · NCT02069756

• Using MRI and biomarkers to monitor muscular dystrophy progression

  This study is testing if MRI and other tests can help track how Duchenne and Becker muscular dystrophies change over time in boys and men, and see how these changes relate to their daily activities and treatments.

  Recruiting at 2 sites University of Florida · Gainesville, Florida and 2 other locations · NCT01484678

NIH-funded research (newest 40 of 50)

Early-stage science funded by the US National Institutes of Health. Most of these projects don't enroll patients, but they show what is being worked on.

• Advancing manufacturing and release testing for a Duchenne gene editing therapy

  A project to optimize how a dual-vector AAV gene editing treatment for Duchenne muscular dystrophy is made and tested, so it can move toward clinical use.

  NIH research Myogene Bio, LLC · San Diego, UNITED STATES · NIH-11526941

• Understanding why Duchenne Muscular Dystrophy worsens and how to stop it

  A research team at USC is studying Duchenne muscular dystrophy to find out why the disease continues to progress and how to interrupt harmful changes in muscle tissue.

  NIH research University of Southern California · Los Angeles, UNITED STATES · NIH-11502706

• Stopping steroid-related muscle and bone wasting in Duchenne muscular dystrophy

  Researchers are testing ways to protect muscles and bones in people with Duchenne muscular dystrophy who take long-term steroid medicines.

  NIH research Univ of Arkansas for Med Scis · Little Rock, United States · NIH-11458914

• MR clues to how nNOS affects energy use and blood flow in Becker and Duchenne muscular dystrophy

  This project looks at how advanced MRI tests can track muscle energy and blood flow changes in people with Becker and Duchenne muscular dystrophy as new gene therapies are explored.

  NIH research University of Florida · Gainesville, United States · NIH-11413866

• Digital motor-skill tracking for infants with Duchenne Muscular Dystrophy using wearables

  A wearable-sensor project aims to quietly track and quantify how motor skills develop in babies and young boys with Duchenne Muscular Dystrophy.

  NIH research Biosensics, LLC · Newton, UNITED STATES · NIH-11407133

• Genetic factors that influence Duchenne Muscular Dystrophy progression

  A study to find genes that change how severe Duchenne muscular dystrophy is in boys and young men, using patient data and DNA samples.

  NIH research Research Inst Nationwide Children's Hosp · Columbus, United States · NIH-11377465

• Optimizing gene therapy to help breathing in Duchenne muscular dystrophy

  A Duke University team is testing a gene therapy approach to improve breathing in boys with Duchenne muscular dystrophy.

  NIH research Duke University · Durham, United States · NIH-11372247

• How immune cells affect the heart in Duchenne muscular dystrophy

  This work looks at how immune cells called macrophages contribute to heart damage in people with Duchenne muscular dystrophy.

  NIH research University of Nevada Reno · Reno, United States · NIH-11372018

• 3D printed human heart model for Duchenne muscular dystrophy (DMD) cardiomyopathy progression

  A patient-friendly 3D-printed heart model using stem cells and gene editing helps study how DMD affects the heart over time and tests potential therapies.

  NIH research University of Minnesota · Minneapolis, United States · NIH-11369821

• Biomarkers and Imaging to Predict Fractures in Duchenne Muscular Dystrophy

  Researchers are testing biochemical and imaging markers to predict which people with Duchenne muscular dystrophy will have fractures, so doctors can prevent them.

  NIH research Johns Hopkins University · Baltimore, United States · NIH-11365265

• Genetic repair of muscle problems in Duchenne muscular dystrophy (DMD)

  Aims to improve muscle and organ function in people with Duchenne muscular dystrophy by testing genetic repair approaches and smooth muscle therapies.

  NIH research Illinois State University · Normal, United States · NIH-11360475

• CRISPR-based gene editing therapy for Duchenne muscular dystrophy using AAV delivery

  A scientist is testing a gene-editing approach to treat Duchenne muscular dystrophy by delivering CRISPR components with an AAV vector to muscle tissue.

  NIH research University of Missouri-Columbia · Columbia, United States · NIH-11351023

• Connexin-43 and Duchenne heart problems: a new target to protect the heart in DMD

  Researchers are studying whether a heart protein called Connexin-43 can be adjusted to help people with Duchenne muscular dystrophy avoid dangerous heart problems.

  NIH research Rutgers Biomedical and Health Sciences · Newark, UNITED STATES · NIH-11335741

• Boosting muscle lymphatics to help Duchenne muscular dystrophy

  Researchers are trying to grow more lymphatic vessels in muscle to lower inflammation and help people with Duchenne muscular dystrophy.

  NIH research Texas A&m University Health Science Ctr · College Station, United States · NIH-11308326

• Predicting heart disease progression in Duchenne and Becker muscular dystrophy

  This project will look for blood tests and heart-imaging signs that predict how heart disease will progress in people with Duchenne or Becker muscular dystrophy and in genetic carriers.

  NIH research Vanderbilt University Medical Center · Nashville, United States · NIH-11252272

• A new RNA treatment to reduce heart scarring in Duchenne muscular dystrophy

  A new RNA-based medicine aims to reduce heart scarring in people with Duchenne muscular dystrophy.

  NIH research Cedars-Sinai Medical Center · Los Angeles, United States · NIH-11252176

• Durable gene edit to restore dystrophin in Duchenne muscular dystrophy

  A gene-editing approach aims to restore dystrophin for people with Duchenne muscular dystrophy by removing DMD exons 45–55 to produce a long-lasting fix.

  NIH research Myogene Bio, LLC · San Diego, UNITED STATES · NIH-11178699

• CRISPR gene editing for Duchenne muscular dystrophy

  This project explores using CRISPR gene technology to correct the genetic cause of Duchenne muscular dystrophy.

  NIH research University of Missouri-Columbia · Columbia, United States · NIH-11175406

• Structure-guided antisense treatment for Duchenne with exon 44 or 45 mutations

  Specially designed antisense molecules aim to boost dystrophin production in people with Duchenne whose genetic mutations can be fixed by skipping exon 44 or 45.

  NIH research University of California Los Angeles · Los Angeles, United States · NIH-11174566

• Understanding genes that change Duchenne Muscular Dystrophy

  This project looks at genetic differences in people with Duchenne muscular dystrophy to understand why the disease affects them differently.

  NIH research Research Inst Nationwide Children's Hosp · Columbus, United States · NIH-11164582

• Understanding how FGF21 affects bone health in Duchenne Muscular Dystrophy

  This study is looking at how a protein called FGF21 might help keep bones healthy for people with Duchenne Muscular Dystrophy (DMD), which can cause muscle loss and bone problems, and it aims to find new ways to prevent bone loss in these patients.

  NIH research University of Iowa · Iowa City, United States · NIH-11161782

• Improving Gene Therapy for Breathing Problems in Duchenne Muscular Dystrophy

  This research aims to make gene therapy better at helping children with Duchenne muscular dystrophy breathe more easily.

  NIH research Duke University · Durham, United States · NIH-11145834

• Biomarker Signatures to Track Duchenne Muscular Dystrophy

  A research project aims to identify and validate blood-based biomarkers that can reliably monitor Duchenne muscular dystrophy progression and response to treatment in people who have the condition.

  NIH research State University of Ny,binghamton · Binghamton, United States · NIH-11134768

• Investigating Connexin 43's role in heart issues related to Duchenne muscular dystrophy

  This study is looking at how a protein called Connexin 43 affects heart health in people with Duchenne muscular dystrophy, with the goal of finding new ways to help prevent heart problems like arrhythmias and heart failure.

  NIH research Rutgers Biomedical and Health Sciences · Newark, UNITED STATES · NIH-11132829

• How fibroblasts may worsen heart damage in Duchenne muscular dystrophy

  This research looks at whether signals from fibroblast cells make heart muscle damage worse in people with Duchenne muscular dystrophy.

  NIH research Indiana University Indianapolis · Indianapolis, United States · NIH-11101331

• Restoring a working, internally shortened dystrophin protein for Duchenne muscular dystrophy

  This project looks at a gene-editing approach delivered by AAV that creates a shortened but working dystrophin to help people with Duchenne muscular dystrophy.

  NIH research Myogene Bio, LLC · San Diego, UNITED STATES · NIH-11088094

• Investigating how lymphatics affect muscle health in Duchenne muscular dystrophy

  This study is looking at how improving the lymphatic system might help reduce inflammation and boost muscle health in people with Duchenne muscular dystrophy (DMD), with hopes of finding new treatments to make life better for those affected by this condition.

  NIH research Texas A&m University Health Science Ctr · College Station, United States · NIH-11067785

• Evaluating a gene editing therapy for Duchenne muscular dystrophy

  This study is testing a new gene editing treatment for Duchenne muscular dystrophy that aims to fix the gene responsible for the condition, and it's designed to help people with this muscle-wasting disease by improving their muscle function over time.

  NIH research Myogene Bio, LLC · San Diego, UNITED STATES · NIH-11066316

• Understanding the role of immune cells in Duchenne muscular dystrophy

  This study is looking at how certain immune cells called macrophages affect muscle inflammation and healing in people with Duchenne muscular dystrophy (DMD), with the hope of finding new ways to help improve treatment for those living with this condition.

  NIH research Hospital for Special Surgery · New York, UNITED STATES · NIH-11053969

• Understanding heart problems in Duchenne and Becker muscular dystrophy

  This study is looking at heart problems that can happen in people with Duchenne and Becker muscular dystrophy to find better ways to check heart health and track how the disease changes over time, which will help improve treatments and future drug trials for those affected.

  NIH research Vanderbilt University Medical Center · Nashville, United States · NIH-11014435

• Investigating a new RNA treatment for heart issues in Duchenne muscular dystrophy

  This study is looking at a new treatment using a special type of RNA called TY4, which comes from tiny particles in the body, to help reduce heart scarring in people with Duchenne muscular dystrophy (DMD), and it’s taken by mouth along with a protein from breast milk to make it easier for patients to use.

  NIH research Cedars-Sinai Medical Center · Los Angeles, United States · NIH-11009962

• A new method to replace defective genes in Duchenne muscular dystrophy

  This study is testing a new treatment for Duchenne muscular dystrophy (DMD) that uses a harmless virus to deliver a healthy version of a gene to help your muscles work better and stay strong.

  NIH research Salk Institute for Biological Studies · La Jolla, UNITED STATES · NIH-11004357

• Investigating how hydrogen sulfide affects Duchenne muscular dystrophy

  This study is looking at how a gas called hydrogen sulfide might help protect muscles in people with Duchenne muscular dystrophy (DMD), a condition that causes muscle weakness, by testing it in animals to find out if it can improve muscle function and reduce damage.

  NIH research University of Minnesota · Minneapolis, United States · NIH-10997019

• Investigating the role of Smad8 in Duchenne muscular dystrophy

  This study is looking at how a protein called Smad8 affects tiny molecules that help muscles work in people with Duchenne muscular dystrophy (DMD), a condition that causes serious muscle loss, to find new ways to help improve muscle health and recovery.

  NIH research University of Alabama at Birmingham · Birmingham, United States · NIH-10993626

• Understanding immune cell interactions in Duchenne muscular dystrophy

  This study is looking at how certain immune cells work together in the muscles of people with Duchenne muscular dystrophy (DMD) to see if changing these interactions can help slow down the disease.

  NIH research University of California-Irvine · Irvine, United States · NIH-10986063

• Improving gene therapy for breathing problems in Duchenne muscular dystrophy

  This study is looking at a new gene therapy to help improve breathing and muscle health for people with Duchenne muscular dystrophy (DMD) by using a special virus to deliver a helpful gene, and it's being tested in mouse models that mimic human conditions.

  NIH research Duke University · Durham, United States · NIH-10980431

• Gene editing therapy to restore dystrophin in Duchenne muscular dystrophy

  This study is testing a new gene editing treatment for Duchenne muscular dystrophy that uses CRISPR technology to fix the genetic problems causing the disease, and it's being done in mice to see how well it works over a longer time, which could lead to better options for patients in the future.

  NIH research Myogene Bio, LLC · San Diego, UNITED STATES · NIH-10938513

• New treatment for heart failure in Duchenne muscular dystrophy patients

  This study is testing a new under-the-skin treatment for heart problems caused by Duchenne muscular dystrophy (DMD) to see if it can help improve heart function and possibly help patients live longer.

  NIH research Revidia Therapeutics, INC · Ellsworth, UNITED STATES · NIH-10913659

• Understanding immune cell interactions in Duchenne muscular dystrophy

  This study is looking at how certain immune cells work together in the muscles of people with Duchenne muscular dystrophy (DMD) to see if understanding this can help find new treatments that might improve their health.

  NIH research University of California-Irvine · Irvine, United States · NIH-10911265

• Improving muscle and bone health in Duchenne Muscular Dystrophy using dietary nitrite.

  This study is looking at how a special dietary supplement called nitrite might help improve muscle and bone health in people with Duchenne Muscular Dystrophy (DMD) by boosting a key molecule that helps with muscle function, especially when there's not enough oxygen.

  NIH research University of Iowa · Iowa City, United States · NIH-10906805

Related topics

Quick answers

How many Duchenne Muscular Dystrophy clinical trials are recruiting right now?

24, as of November 12, 2026. We update this list each time we refresh our data from ClinicalTrials.gov.

Where are these trials running?

In 26 countries. The most sites are in United States (15 trials), Belgium (5 trials), United Kingdom (5 trials), Italy (4 trials), Canada (4 trials). Each study page lists its sites and which ones are recruiting.

What phases are they in?

Phase 2: 4, Phase 4: 2, Phase 1: 2, Phase 1/2: 2. Trials with no phase listed are usually device, behavioural or observational studies.

How do I join one?

Open a study, check the "Is this trial a fit for you?" section, then contact the study team listed on the page. They will ask a few screening questions. Asking is free and does not commit you to anything. Talk it over with your doctor first.

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