Condition

Myotonic Dystrophy clinical trials and research

• 12Clinical trials
• 43NIH research projects
• 16Countries

Newest Myotonic Dystrophy clinical trials

All recruiting trials (12)

• Understanding long-term progression in myotonic dystrophy type 1 (DM1)

  This study will follow adults with DM1 for at least four years to see how walking, hand function, muscle strength, heart rhythms, and lung function change over time.

  Recruiting Virginia Commonwealth University · Richmond, Virginia · NCT07700225

• Zeleciment Basivarsen (DYNE-101) for people with myotonic dystrophy type 1

  This trial will test whether Zeleciment Basivarsen (DYNE-101) improves symptoms and is safe and tolerable for adults with myotonic dystrophy type 1.

  Recruiting at 44 sites Phase 3 Dyne Therapeutics · La Jolla, California and 43 other locations · NCT07486934

• Quantitative MRI measurement of muscle changes in myotonic dystrophy

  This project will test whether advanced quantitative MRI scans can measure muscle structure and link to disease severity and RNA changes in adults with myotonic dystrophy.

  Recruiting Wake Forest University Health Sciences · Winston-Salem, North Carolina · NCT07362875

• 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

• SAR446268 gene therapy for non-congenital myotonic dystrophy type 1

  This will try a single intravenous dose of SAR446268 gene therapy to lower toxic DMPK mRNA and improve muscle function in people aged 10 to 55 with non‑congenital myotonic dystrophy type 1.

  Recruiting at 10 sites Phase 1/2 Sanofi · Gainesville, Florida and 9 other locations · NCT06844214

• Assessing Readiness and Endpoints for Pediatric Myotonic Dystrophy

  This study is trying to improve how we understand and measure the effects of congenital and childhood myotonic dystrophy to help create better treatments for kids with these conditions.

  Recruiting Virginia Commonwealth University · Little Rock, Arkansas and 3 other locations · NCT06747884

• Once-daily prolonged-release mexiletine for myotonic dystrophy types 1 and 2 (HERCULES)

  This study will test whether once-daily prolonged-release mexiletine can reduce myotonia in people aged 16 and older with genetically confirmed myotonic dystrophy type 1 or 2.

  Recruiting at 7 sites Phase 3 Lupin Ltd. · Leuven and 6 other locations · NCT06523400

• Non-invasive prenatal diagnosis for single gene disorders

  This study is testing a new way to safely diagnose single-gene disorders in unborn babies using a simple blood test from the mother, starting as early as 9 weeks into the pregnancy.

  Recruiting Assistance Publique - Hôpitaux de Paris · Paris · NCT06147414

• 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

• National registry for myotonic dystrophy in the UK

  This study is setting up a registry to gather information from people with myotonic dystrophy to help improve future treatments and find participants for clinical trials.

  Recruiting Newcastle University · Newcastle upon Tyne · NCT04003363

• Online registry for myotonic dystrophy patients

  This study is creating an online registry for people with myotonic dystrophy to share their experiences and help researchers find better treatments.

  Recruiting Myotonic Dystrophy Foundation · Oakland, California · NCT02398786

• 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

NIH-funded research (newest 40 of 43)

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.

• New small molecule therapeutics to help myotonic dystrophy

  A new NIH-funded project tests specially designed small molecules to lower harmful RNA activity in myotonic dystrophy and related diseases, aiming to reduce disease-driving RNA effects.

  NIH research State University of New York at Albany · Albany, United States · NIH-11617693

• Mechanisms of muscle disease in myotonic dystrophy type 1

  This project looks at why skeletal muscles weaken and waste away in adults with myotonic dystrophy type 1 and aims to uncover the underlying molecular causes.

  NIH research Baylor College of Medicine · Houston, United States · NIH-11597907

• Extracellular vesicle biomarkers to monitor DM1 using urine and blood samples

  A study looking for easy, noninvasive biomarkers in urine and blood to track disease activity in people with myotonic dystrophy type 1.

  NIH research Massachusetts General Hospital · Boston, United States · NIH-11494099

• Calcium Channel Blocker in DM1: Can amlodipine help myotonic dystrophy type 1

  This study tests whether the calcium‑channel blocker amlodipine can improve symptoms and survival in myotonic dystrophy type 1 (DM1).

  NIH research University of Rochester · Rochester, United States · NIH-11444061

• Preparing therapies for DM1 across ages, with a focus on children

  A broad, patient-focused effort to bring promising DM1 therapies to both children and adults by enriching trial readiness, biomarkers, and natural history data.

  NIH research Virginia Commonwealth University · Richmond, United States · NIH-11423652

• Biological factors that drive variability in myotonic dystrophy

  This grant explores why myotonic dystrophy can look very different from person to person by studying its genetic changes and how they affect muscle in adults.

  NIH research University of Florida · Gainesville, United States · NIH-11404431

• Identifying RNA Splicing and Gene Expression Changes in congenital myotonic dystrophy

  This grant looks at how RNA changes contribute to congenital myotonic dystrophy to understand why symptoms change as children grow.

  NIH research Virginia Commonwealth University · Richmond, United States · NIH-11393159

• Understanding why skeletal muscles weaken in adult myotonic dystrophy type 1

  Researchers are studying how a faulty RNA in myotonic dystrophy type 1 damages muscle, aiming to uncover why muscles weaken and waste away in adults with the disease.

  NIH research Baylor College of Medicine · Houston, United States · NIH-11389442

• Understanding why muscles weaken in myotonic dystrophy type 1

  This project aims to find what causes progressive muscle weakness in people with myotonic dystrophy type 1 by studying how abnormal RNA affects muscle cells.

  NIH research Baylor College of Medicine · Houston, United States · NIH-11387396

• Extracellular RNA biomarkers in myotonic dystrophy

  A study at a major hospital will look for extracellular RNA in body fluids to find clues about myotonic dystrophy and how it affects the brain and muscles.

  NIH research Massachusetts General Hospital · Boston, United States · NIH-11379699

• How Calcium Dysregulation in Skeletal Muscle Affects Myotonic Dystrophy

  This research looks at why skeletal muscles misbehave in myotonic dystrophy and how correcting calcium signaling might improve muscle strength and breathing.

  NIH research University of Rochester · Rochester, United States · NIH-11357592

• Modeling DM1 and DM2 Brain Disease with patient-derived brain organoids

  Scientists will use patient-derived stem cells to grow brain-like organoids to study why DM1 and DM2 affect the brain and behavior.

  NIH research Regenerative Research Foundation · Albany, UNITED STATES · NIH-11356069

• Biomarkers and outcome measures for Myotonic Dystrophy Type 1

  The project aims to find blood and clinical signs that track disease course and treatment effects in people with Myotonic Dystrophy Type 1.

  NIH research Virginia Commonwealth University · Richmond, United States · NIH-11349342

• Longitudinal brain and biomarker study in myotonic dystrophy type 2

  A long-term study of brain structure, cognitive symptoms, and lab biomarkers in adults with myotonic dystrophy type 2 to understand how the disease affects the brain over time.

  NIH research Wake Forest University Health Sciences · Winston-Salem, United States · NIH-11347603

• Extracellular RNA biomarkers to monitor myotonic dystrophy type 1

  Doctors are studying urine RNA signals that may reflect how DM1 affects muscle function, to help track disease activity without painful biopsies.

  NIH research Massachusetts General Hospital · Boston, United States · NIH-11345157

• New small molecules to help treat myotonic dystrophy

  A new NIH grant is developing small molecule drugs to help people with myotonic dystrophy feel and function better.

  NIH research Repeat RNA Therapeutics INC · Menands, United States · NIH-11315150

• Small-molecule medicines targeting myotonic dystrophy

  New small-molecule medicines aim to lower the toxic RNAs that cause symptoms in people with myotonic dystrophy.

  NIH research State University of New York at Albany · Albany, United States · NIH-11307593

• Small-molecule treatments for myotonic dystrophy type 1

  Developing new small-molecule drugs that target the toxic RNA that causes myotonic dystrophy type 1 for people living with DM1.

  NIH research University of Southern California · Los Angeles, UNITED STATES · NIH-11263628

• RNA splicing and gene expression changes in congenital myotonic dystrophy

  This project looks at how changes in RNA splicing and gene activity lead to congenital myotonic dystrophy in infants, children, and adolescents.

  NIH research Virginia Commonwealth University · Richmond, United States · NIH-11179128

• How muscles are damaged in myotonic dystrophy type 1

  This project will uncover how abnormal RNA and protein changes cause muscle weakness and wasting in people with myotonic dystrophy type 1.

  NIH research Baylor College of Medicine · Houston, United States · NIH-11164817

• RNA markers for myotonic dystrophy in spinal fluid and blood

  This project looks for RNA signals in spinal fluid and blood that could help track brain and muscle changes in people with myotonic dystrophy type 1.

  NIH research Massachusetts General Hospital · Boston, United States · NIH-11162389

• Why myotonic dystrophy symptoms and timing differ between people

  Researchers will look at repeat lengths and other biological signs in adults with myotonic dystrophy to help explain why symptoms and age of onset vary.

  NIH research University of Florida · Gainesville, United States · NIH-11161633

• Investigating brain structure and cognitive function in myotonic dystrophy type 2.

  This study is looking at how myotonic dystrophy type 2 affects the brain and thinking skills in adults, and it aims to gather information that could help us understand the memory and thinking challenges that come with this condition.

  NIH research Wake Forest University Health Sciences · Winston-Salem, United States · NIH-11116011

• Understanding Muscle Weakness in Myotonic Dystrophy

  This research aims to understand why muscles weaken in Myotonic Dystrophy and explore if an existing medication could help.

  NIH research University of Rochester · Rochester, United States · NIH-11101114

• Using AI and patient-derived lab-grown cells to find treatments for myotonic dystrophy

  This project combines artificial intelligence with patient-derived stem-cell models to speed discovery of safer, more effective medicines for adults with myotonic dystrophy type 1.

  NIH research Greenstone Biosciences, INC. · Palo Alto, United States · NIH-11098495

• Understanding muscle problems in myotonic dystrophy type 1

  This study is looking at how certain genetic changes in people with myotonic dystrophy type 1 can cause muscle weakness and wasting, and it aims to find new ways to help manage these issues by understanding how specific RNA molecules affect muscle function.

  NIH research Baylor College of Medicine · Houston, United States · NIH-11075579

• Developing new small molecule treatments for myotonic dystrophy

  This study is working on new medicines to help people with myotonic dystrophy by creating special compounds that can target and reduce the harmful RNA causing the condition, with the hope of easing symptoms and improving lives.

  NIH research State University of New York at Albany · Albany, United States · NIH-11060026

• Identifying biomarkers and clinical endpoints for myotonic dystrophy type-1

  This study is looking at myotonic dystrophy type-1, a common muscle disease in adults, to find better ways to measure how severe the condition is and how well new treatments might work, so we can help improve care for people living with this condition.

  NIH research Virginia Commonwealth University · Richmond, United States · NIH-11058441

• New treatments for myotonic dystrophy type 1

  This study is looking for new medicines to help people with myotonic dystrophy type 1 by finding special compounds that can fix the problems caused by a faulty gene, and so far, they’ve found one that shows promise in improving symptoms.

  NIH research University of Southern California · Los Angeles, UNITED STATES · NIH-11012267

• Understanding how CELF1 contributes to Myotonic Dystrophy Type 1

  This study is looking at a protein called CELF1 that is linked to muscle weakness in people with Myotonic Dystrophy Type 1, using special mice to learn how it affects muscle function, which could help find new ways to treat the condition.

  NIH research Baylor College of Medicine · Houston, United States · NIH-10996098

• Investigating the factors that affect the severity of myotonic dystrophy.

  This study is looking at how certain genetic differences affect the symptoms of myotonic dystrophy types 1 and 2, and it’s designed for patients who want to learn more about how their genes might influence their condition, all while participating from home.

  NIH research University of Florida · Gainesville, United States · NIH-10992318

• Identifying non-invasive biomarkers for myotonic dystrophy

  This study is looking for easy and safe ways to track how myotonic dystrophy changes over time and how well new treatments are working, so that people with this condition can get better care.

  NIH research Massachusetts General Hospital · Boston, United States · NIH-10991811

• Understanding how MBNL loss affects smooth muscle in myotonic dystrophy type 1

  This study is looking into how myotonic dystrophy type 1 affects the digestive system, especially problems like trouble swallowing and constipation, by exploring what happens when a specific protein is not working properly in the muscles of the gut, using mice to help understand these issues better.

  NIH research Baylor College of Medicine · Houston, United States · NIH-10975935

• Understanding the genetic causes of Myotonic Dystrophy Type 2

  This study is looking at Myotonic Dystrophy Type 2, a genetic disorder, by using yeast to understand how certain DNA repeats can cause problems in our genes, which might help us find new ways to treat this condition.

  NIH research California State University San Marcos · San Marcos, United States · NIH-10975184

• Identifying RNA biomarkers in myotonic dystrophy

  This study is looking at myotonic dystrophy to see if certain RNA patterns in spinal fluid can help us understand how the disease is changing over time and how well treatments are working, which could lead to better ways to diagnose and treat patients like you.

  NIH research Massachusetts General Hospital · Boston, United States · NIH-10931559

• Understanding muscle problems in myotonic dystrophy type 1

  This study is looking into why people with myotonic dystrophy type 1 experience muscle weakness and wasting, focusing on how a specific genetic change affects muscle cells, and it hopes to find new ways to help treat this condition by working with patients to collect blood and muscle samples.

  NIH research Baylor College of Medicine · Houston, United States · NIH-10922861

• Investigating brain structure and function in myotonic dystrophy type 2

  This study is looking at how the brain's structure and function affect thinking and movement in people with myotonic dystrophy type 2, hoping to better understand the cognitive challenges you might face, so we can improve treatments for those issues.

  NIH research Wake Forest University Health Sciences · Winston-Salem, United States · NIH-10894728

• Understanding the RNA changes that lead to congenital myotonic dystrophy

  This study is looking at how congenital myotonic dystrophy affects kids' muscles from birth to age 16 by examining muscle samples to see how changes in genes relate to their symptoms, with the hope of finding better ways to diagnose and treat the condition.

  NIH research Virginia Commonwealth University · Richmond, United States · NIH-10882023

• Investigating RNA biomarkers in myotonic dystrophy type 1

  This study is looking for helpful markers in urine that can show how myotonic dystrophy type 1 is affecting your muscles, making it easier to track the disease without needing a muscle biopsy.

  NIH research Massachusetts General Hospital · Boston, United States · NIH-10856448

• Investigating how RNA processing affects brain function in myotonic dystrophy

  This study is looking at how problems with RNA processing in the brain can affect people with myotonic dystrophy, and it hopes to find new ways to help improve brain function for those living with this condition.

  NIH research University of Florida · Gainesville, United States · NIH-10852078

Related topics

Quick answers

How many Myotonic 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 16 countries. The most sites are in United States (8 trials), United Kingdom (4 trials), France (3 trials), Australia (2 trials), Belgium (2 trials). Each study page lists its sites and which ones are recruiting.

What phases are they in?

Phase 3: 2, Phase 1/2: 1. 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

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