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Neurological & Neuromuscular

Myotonic Dystrophy (DM1) Clinical Trials and Treatments

Also called DM1, DM2, Dystrophia Myotonica, Steinert Disease

Myotonic dystrophy is caused by a repeat expansion in the DMPK gene (DM1, a 3-letter CTG repeat) or the CNBP gene (DM2, a 4-letter CCTG repeat). In DM1, a CTG repeat expands in the 3' untranslated region, while DM2 involves CCTG repeats. The pathophysiology involves toxic accumulation of mutant mRNA, which sequesters RNA-binding proteins and disrupts normal RNA splicing and protein production.

In plain words, myotonic dystrophy is a genetic muscle condition that causes weakness over time and muscles that are slow to relax after use. Beyond the muscles, it can also affect the heart and eyes as well as the brain and hormone system.

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Myotonic Dystrophy

Where treatments stand, from ClinicalTrials.gov and FDA labels

Open and active trials35
FDA review0

No decision pending. FDA calendar

FDA-approved medicines0

No medicine's current FDA label names Myotonic Dystrophy yet.

Myotonic Dystrophy has no FDA-approved medicines, 35 open or active clinical trials, and no pending FDA decision.

Labels checked October 21, 2026.Download the data

Latest for Myotonic Dystrophy

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More about Myotonic Dystrophy

This leads to faulty versions of many proteins, including the muscle chloride channel, whose loss causes myotonia. The repeat number correlates with disease severity and age of onset, with longer repeats generally causing more severe disease with earlier presentation.

Keep reading about Myotonic Dystrophy

DM1 shows two main phenotypes: classic form with adult onset and congenital form with severe neonatal presentation and intellectual disability. DM2 generally has milder symptoms than DM1. Beyond skeletal muscle, myotonic dystrophy affects smooth muscle, cardiac muscle, and the brain. Cardiac involvement ranges from conduction abnormalities to arrhythmias and dilated cardiomyopathy.

Cerebral manifestations include intellectual disability in congenital forms, behavioral changes, cognitive decline, and increased risk of central sleep apnea. Cataracts are common in DM1 and can also occur in DM2. In mild DM1 they often appear in mid to late adulthood. Endocrine dysfunction includes testicular atrophy, insulin resistance, and thyroid abnormalities.

What are the symptoms of Myotonic Dystrophy?

These are common symptoms of Myotonic Dystrophy, and how strong they are varies from person to person.

  • Progressive muscle weakness and wasting
  • Myotonia causing stiff muscles that relax slowly after use
  • Facial drooping and weakness affecting chewing and swallowing
  • Ptosis and cataracts affecting vision
  • Cognitive and behavioral changes
  • Cardiac arrhythmias and conduction abnormalities

Who gets Myotonic Dystrophy?

Myotonic Dystrophy Type 1 (DM1) typically appears in adolescence to adulthood, though congenital forms occur in infants. Myotonic Dystrophy Type 2 (DM2) typically appears in adulthood. Affects males and females equally. Autosomal dominant inheritance with anticipation—children often have more severe disease and earlier onset. Occurs across all populations.

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Which new Myotonic Dystrophy medicines are in trials?

Investigational medicines Trial Friend tracks for Myotonic Dystrophy, with trial results, safety findings and where each one stands.

Zeleciment basivarsenPhase 3
Antibody-conjugated antisense oligonucleotide · Dyne Therapeutics

How to get help paying for Myotonic Dystrophy treatment

Charity funds and drugmaker programs for Myotonic Dystrophy, checked at the source.

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Charity funds
  • From a charity · Muscular Dystrophy Association
    MDA Durable Medical Equipment (DME) Grant Program fund
    Apply directly

    Pays for: Medical equipment (wheelchairs, lifts, canes and other DME), up to $1,000 per year.

    The foundation says: “Status not shown on page”
Status as each foundation showed it on November 9, 2026.

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Side effects people have reported to the FDA

Side effects people have reported to the FDA

Real-world side effect reports from the FDA Adverse Event Reporting System (FAERS). Includes both FDA-approved drugs and investigational therapies from active clinical trials. Click any drug to see what patients reported.

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Diagnosis and genetic testing

Genetic testing for Myotonic Dystrophy

Genetic testing can confirm a diagnosis, guide treatment decisions, and identify family members who may be at risk.

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Trusted Myotonic Dystrophy resources

Reputable organizations and medical references for learning more about Myotonic Dystrophy, including disease registries, foundation resources, and clinical guidelines.

Active clinical trials for Myotonic Dystrophy

Use this Myotonic Dystrophy clinical trial finder to see the 28 studies recruiting patients and 2 not yet recruiting in the United States and worldwide, with eligibility criteria in plain English. Of these, 14 test a treatment and 16 are observational studies or registries; each card says which. Every trial comes from ClinicalTrials.gov.

Jump to the trial list, the site map or the pipeline timeline.

Note: Trial recruitment statuses on ClinicalTrials.gov may not immediately reflect recent FDA decisions, sponsor announcements, or enrollment changes. Always confirm a trial's current status directly with the study coordinator before making plans.

35 active trials worldwide
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RecruitingTreatment trialPhase 3Recently updatedNCT07486934

Efficacy, Safety, and Tolerability of Zeleciment Basivarsen (DYNE-101) in Participants With Myotonic Dystrophy Type 1

Intervention: zeleciment basivarsen (DYNE-101), Placebo

Sponsor: Dyne Therapeutics

The purpose of the study is to assess the efficacy, safety, and tolerability of zeleciment basivarsen (DYNE-101) for the treatment of myotonic dystrophy 1 (DM1).

Ages 16 Years+
Started May 2026Updated todayEst. Jul 2028 (~1y 8m)
RecruitingTreatment trialPhase 3Recently updatedNCT07587242

A Phase 3 Study to Evaluate the Safety and Efficacy of AOC 1044 (Also Referred to as Delpacibart Zotadirsen) in Participants With DMD With Gene Mutations Amenable to Exon 44 Skipping

Intervention: AOC 1044, Placebo

Sponsor: Avidity Biosciences, Inc.

A Randomized, Double-blind, Placebo-controlled, Phase 3 Study to Evaluate the Efficacy and Safety of Intravenous AOC 1044 for the treatment of Duchenne Muscular Dystrophy (DMD) with Gene Mutations Amenable to Exon 44 Skipping

Ages 7 Years to 16 Years
Started Sep 2026Updated 3 days agoEst. May 2029 (~2y 6m)
Not yet recruitingTreatment trialPhase 1Recently updatedNCT07075965

Calcium Channel Blocker in Myotonic Dystrophy Type 1

Intervention: Amlodipine

Sponsor: University of Rochester

Funding Source - FDA OOPD

Ages 18 Years to 65 Years
Expected to start Dec 2026Updated 4 days agoEst. Oct 2030 (~3y 10m)
RecruitingObservational study, no treatment givenRecently updatedNCT05224778

DMCRN-02-001: Assessing Pediatric Endpoints in DM1

Sponsor: Virginia Commonwealth University

The overall goal of the study is to establish valid clinical endpoint assessments for children with congenital myotonic dystrophy type 1 and develop biomarkers for the condition.

Ages up to 59 Months
Started Aug 2022Updated 1 week agoEst. Dec 2026 (~1 month)
RecruitingObservational study, no treatment givenRecently updatedNCT07912983

Metabolomic Biomarkers of Myotonic Dystrophy

Sponsor: Massachusetts General Hospital

Current methods of measuring the response to new treatments for muscular dystrophies involve the examination of small pieces of muscle tissue called biopsies. The investigators are interested in finding less invasive methods that reduce the need for muscle biopsies. The purpose o...

Ages 14 Years+1 location
Started Oct 2026Updated 1 week agoEst. Aug 2030 (~3y 9m)
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Active trial locations29 cities in the US
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Trial Pipeline

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Data from ClinicalTrials.gov, U.S. National Library of Medicine.
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Myotonic Dystrophy patient communities

Facebook groups, Reddit communities and YouTube channels where Myotonic Dystrophy patients, caregivers and advocacy groups talk.

Myotonic Dystrophy Support
Patient and caregiver community
r/MyotonicDystrophy
Myotonic dystrophy community
NORD Rare Disease Report
Reviewed plain-language medical overview from the National Organization for Rare Disorders, with assistance program listings

Companies developing Myotonic Dystrophy treatments

Drugmakers that list Myotonic Dystrophy in their rare disease portfolio, 7 in all.

Dyne Therapeutics
Rare disease specialist · 3 rare diseases
Novartis
Diversified pharma · 59 rare diseases
Vertex Pharmaceuticals
Rare disease specialist · 12 rare diseases
Sarepta Therapeutics
Rare disease specialist · 5 rare diseases
Avidity Biosciences
Rare disease specialist · 3 rare diseases
Sanofi / Genzyme
Diversified pharma · 27 rare diseases
Ionis Pharmaceuticals
Diversified pharma · 20 rare diseases

Questions people ask about Myotonic Dystrophy