Condition

Muscular Dystrophy clinical trials and research

• 12Clinical trials
• 114NIH research projects
• 4Countries

Newest Muscular Dystrophy clinical trials

All recruiting trials (12)

• 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

• Amino acids plus diet and exercise for people with FSHD

  This trial will test whether taking essential amino acid supplements together with a structured diet and exercise program helps adults with FSHD build muscle, reduce fat, and improve physical performance compared with placebo and matched healthy adults.

  Recruiting University of Pavia · Voghera, Pavia · NCT07543016

• Portable markerless video system to measure walking in adults with neuromuscular conditions or healthy volunteers.

  This project will test a portable video device that measures walking patterns to see if it can track gait changes in adults with neuromuscular diseases and in healthy volunteers.

  Recruiting Institut de Myologie, France · Paris · NCT07321977

• 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

• Using a robotic exoskeleton to help children with movement disorders walk better

  This study is testing if a robotic exoskeleton can help children with movement disorders walk better and improve their overall movement skills.

  Recruiting National Institutes of Health Clinical Center (CC) · Bethesda, Maryland · NCT05726591

• Evaluating the Nomad powered KAFO for improving mobility in individuals with lower-extremity impairments

  This study is testing if a new powered brace can help people with leg injuries move better and feel more balanced compared to their regular braces.

  Recruiting Shirley Ryan AbilityLab · Chicago, Illinois · NCT05644522

• Using ultrasound to study nerves and muscles

  This study is testing how ultrasound can help us learn more about the health of nerves and muscles in both healthy adults and those with neuromuscular disorders.

  Recruiting National Institutes of Health Clinical Center (CC) · Bethesda, Maryland · NCT05237973

• Feasibility of a brain-computer interface for people with tetraplegia

  This study is testing a new brain-computer interface that helps people with tetraplegia control a computer and other devices just by thinking, to see if it’s safe and effective.

  Recruiting at 6 sites Massachusetts General Hospital · Sacramento, California and 5 other locations · NCT00912041

• Understanding early signs of dystroglycanopathies in muscular dystrophy

  This study is looking to find out the early signs and symptoms of dystroglycanopathies in people with muscular dystrophy to help improve future treatments and care.

  Recruiting University of Iowa · Iowa City, Iowa · NCT00313677

• Exploring the genetics behind heart and muscle diseases

  This study is trying to find out how genetics play a role in heart and muscle diseases by looking at blood samples from families with these conditions.

  Recruiting University of Chicago · Chicago, Illinois · NCT00138931

• Registry for Myotonic Dystrophy and Facioscapulohumeral Muscular Dystrophy

  This study is creating a registry for people with myotonic dystrophy or facioscapulohumeral muscular dystrophy to share their health information and connect with researchers working to learn more about these conditions.

  Recruiting University of Rochester · Rochester, New York · NCT00082108

• Understanding inherited neurological disorders

  This study is trying to learn more about inherited neurological disorders by looking at the medical history and genetics of people who have or might have these conditions.

  Recruiting National Institutes of Health Clinical Center (CC) · Bethesda, Maryland and 1 other locations · NCT00004568

NIH-funded research (newest 40 of 114)

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

• Endocrine role of intramuscular fat in muscular dystrophy

  This grant studies how fat that builds up inside muscles in Duchenne and Becker muscular dystrophy may release hormones that affect overall health, especially metabolism.

  NIH research University of Florida · Gainesville, United States · NIH-11460264

• 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

• Curation of muscular dystrophy genes to improve diagnosis and care

  A grant to help doctors better interpret genetic tests for muscular dystrophy and myopathy so patients get clearer diagnoses and faster access to therapies.

  NIH research Washington University · Saint Louis, United States · NIH-11417940

• 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

• Muscular Dystrophy Center focused on DM1/DM2 therapies

  A Wellstone Center at UF/UR is researching transformative therapies for myotonic dystrophy types 1 and 2, with strong collaboration between basic scientists and clinicians to speed up potential treatments.

  NIH research University of Florida · Gainesville, United States · NIH-11404417

• Muscular Dystrophy Specialized Research Center (P50)

  A long-running research program at the University of Iowa is studying the natural course of dystroglycanopathies to improve care and guide future trials for people of all ages with FKRP-related and other gene mutations.

  NIH research University of Iowa · Iowa City, United States · NIH-11402658

• Muscular Dystrophy Center research to understand breathing problems in dystroglycanopathy

  A specialized NIH grant is funding a center to understand why breathing problems occur in people with dystroglycanopathies and to uncover the cellular causes behind these muscle and respiratory issues.

  NIH research University of Iowa · Iowa City, United States · NIH-11402651

• Muscular Dystrophy Center for Training and Discovery

  A specialized NIH grant supports training the next generation of doctors, scientists, and clinicians to advance muscular dystrophy care and research.

  NIH research University of Iowa · Iowa City, United States · NIH-11402642

• Muscular Dystrophy Center Focus: tissue banks, cell culture and diagnostics

  A national muscular dystrophy center is creating and sharing muscle tissue, cell lines, and advanced diagnostic tests to accelerate research and improve patient care.

  NIH research University of Iowa · Iowa City, United States · NIH-11402636

• Muscular Dystrophy Center to Help Patients Live Better

  This grant supports an Administrative Core at a muscular dystrophy center to coordinate research, share information, and engage patients and families.

  NIH research University of Iowa · Iowa City, United States · NIH-11402633

• Muscular Dystrophy Specialized Research Center at UIowa

  A large, patient-focused program is studying muscular dystrophy caused by problems with a protein called α-dystroglycan to improve muscle function and breathing.

  NIH research University of Iowa · Iowa City, United States · NIH-11402631

• Understanding how fibroblasts influence muscular dystrophy progression

  This project studies how muscle-support cells interact with damaged muscle to drive disease in muscular dystrophy, using a mouse model to uncover new therapeutic targets for patients.

  NIH research Cincinnati Childrens Hosp Med Ctr · Cincinnati, United States · NIH-11401970

• Muscular Dystrophy Cell Line and Serum Banking Core

  A dedicated resource that creates and stores patient-derived muscle-related cell lines and blood samples to speed the development of genetic therapies for muscular dystrophy.

  NIH research Research Inst Nationwide Children's Hosp · Columbus, United States · NIH-11397447

• MDSRC for Next Directions in Muscular Dystrophy Research

  A major research center grant aims to speed new gene therapy ideas for muscular dystrophy by sharing expertise, creating tools, and advancing three focused projects.

  NIH research Research Inst Nationwide Children's Hosp · Columbus, United States · NIH-11397440

• Muscular dystrophy gene therapy center in Seattle

  A Seattle-based program studies gene therapy approaches to muscular dystrophy to help patients in the Northwest and beyond.

  NIH research University of Washington · Seattle, United States · NIH-11395621

• Clinical Trial Readiness for Very Young Children with LAMA2-Related Muscular Dystrophy

  Aims to prepare very young children with LAMA2-related muscular dystrophy for future therapies by validating measurements and biomarkers that can track progress.

  NIH research Research Inst Nationwide Children's Hosp · Columbus, United States · NIH-11393814

• Muscle-targeted RNA therapeutics platform for muscular dystrophy

  A patient-friendly program aims to deliver therapeutic RNA molecules directly to muscle cells to treat inherited neuromuscular disorders like facioscapulohumeral muscular dystrophy.

  NIH research Mirecule, INC. · Gaithersburg, UNITED STATES · NIH-11393057

• Cardiac TP receptor blocking to protect the heart in Duchenne muscular dystrophy

  Aims to see if blocking the thromboxane-prostanoid receptor helps prevent heart damage in people with Duchenne muscular dystrophy.

  NIH research Vanderbilt University Medical Center · Nashville, United States · NIH-11391058

• Emerin self-assembly at the nuclear envelope and Emery-Dreifuss muscular dystrophy

  Aims to understand how emerin proteins organize themselves at the nuclear envelope to protect muscle cell nuclei, with the goal of helping people with Emery-Dreifuss muscular dystrophy.

  NIH research University of Southern California · Los Angeles, UNITED STATES · NIH-11380607

• 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

• Molecular basis of a rare LMNA-related muscle disease and potential gene-editing therapy

  A small NIH-funded project will study how a specific LMNA mutation causes early muscle weakness and explore a potential correction with a gene-editing approach using patient cells.

  NIH research Univ of Maryland, College Park · College Park, United States · NIH-11374055

• 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

• C2 domain therapies for muscular dystrophy

  A patient-friendly look at a lab-driven approach to help people with dysferlin-related muscular dystrophy through targeted C2 domain proteins.

  NIH research University of Maryland Baltimore · Baltimore, United States · NIH-11345565

• 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

• Metabolic imaging to detect early muscle and heart changes in Duchenne and Becker muscular dystrophy

  Using advanced metabolic imaging to find early muscle and heart changes in people with Duchenne or Becker muscular dystrophy.

  NIH research Ut Southwestern Medical Center · Dallas, United States · NIH-11333270

• Laminin protein treatment for LAMA2 (MDC1A) congenital muscular dystrophy

  This project aims to develop a laminin protein therapy to slow muscle weakness in people with LAMA2 (MDC1A) congenital muscular dystrophy.

  NIH research University of Nevada Reno · Reno, United States · NIH-11314568

• Mitochondria, calcium, and muscle cell death in muscular dystrophy

  Researchers are exploring whether changing how mitochondria handle calcium in muscle cells can reduce muscle cell death in people with muscular dystrophy.

  NIH research Baylor College of Medicine · Houston, United States · NIH-11312628

• 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

• TRPC6 blocker to protect the heart in Duchenne muscular dystrophy

  This project uses a drug that blocks the TRPC6 channel to protect heart and skeletal muscles in adults with Duchenne muscular dystrophy.

  NIH research Medical College of Wisconsin · Milwaukee, United States · NIH-11291309

• 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

• Using mineralocorticoid receptor blockers to reduce inflammation in Duchenne muscular dystrophy

  This project sees if drugs that block mineralocorticoid receptors can lower muscle inflammation and help AAV gene therapy work better for people with Duchenne muscular dystrophy.

  NIH research Ohio State University · Columbus, UNITED STATES · NIH-11251255

Related topics

Quick answers

How many Muscular Dystrophy clinical trials are recruiting right now?

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

Where are these trials running?

In 4 countries. The most sites are in United States (10 trials), Mali (1 trial), France (1 trial), Italy (1 trial). Each study page lists its sites and which ones are recruiting.

What phases are they in?

Phase 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.

Find the studies that fit you

A list is a starting point. Tell us your situation in your own words and we'll rank the studies that match and explain why. It's free and needs no account.

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