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Neurological & Neuromuscular

Becker Muscular Dystrophy (BMD) Clinical Trials

Also called BMD, Benign Duchenne-type Muscular Dystrophy

Becker Muscular Dystrophy results from mutations in the dystrophin gene on the X chromosome that produce an internally deleted or abnormal but partially functional dystrophin protein. In contrast to DMD mutations causing complete dystrophin absence or non-functional protein, BMD mutations preserve the reading frame and allow production of shorter but partially active dystrophin. The amount of residual dystrophin function correlates with disease severity and age of onset. In-frame mutations generally produce milder phenotypes than out-of-frame mutations, though genotype-phenotype correlation is not absolute.

In plain words, Becker muscular dystrophy is an inherited muscle condition where the body makes a muscle protein called dystrophin that only partly works. Muscles slowly weaken, starting in the hips and thighs, and the heart muscle is often affected too.

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Becker Muscular Dystrophy

Where treatments stand, from ClinicalTrials.gov and FDA labels

Also 18 studies with no phase, like observational studies.

FDA review0

No decision pending. FDA calendar

FDA-approved medicines0

No medicine's current FDA label names Becker Muscular Dystrophy yet.

Becker Muscular Dystrophy has no FDA-approved medicines, 18 open or active clinical trials (13 recruiting), and no pending FDA decision.

Labels checked October 21, 2026.Download the data

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Pathophysiology involves incomplete dystrophin deficiency leading to progressive muscle fiber damage, necrosis, inflammation, and replacement by fat and connective tissue. Muscles affected include skeletal muscles (progressive weakness), cardiac muscle (cardiomyopathy, which is common and becomes more likely with age), and rarely respiratory muscles. BMD shows slower progression than DMD, with some patients retaining ambulation into the third to fourth decade of life.

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Serum creatine kinase levels are markedly elevated. Complications include respiratory insufficiency in later stages, cardiac arrhythmias and heart failure, rhabdomyolysis with myoglobinuria following intense activity or trauma, and contractures limiting mobility.

What are the symptoms of Becker Muscular Dystrophy?

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

  • Progressive muscle weakness starting in hip and thigh muscles
  • Calf hypertrophy and muscle wasting in other areas
  • Waddling gait and difficulty climbing stairs
  • Myoglobinuria causing dark urine after intense activity
  • Cramps and muscle pain
  • Cardiomyopathy (heart muscle disease), which is common and becomes more likely with age

Who gets Becker Muscular Dystrophy?

Primarily affects males; females are usually carriers with minimal or no symptoms. Typical onset between ages 5 and 15 years, though can vary widely (5 to 60 years). X-linked recessive inheritance pattern. Occurs in all ethnic backgrounds.

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Which new Becker Muscular Dystrophy medicines are in trials?

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

How to get help paying for Becker Muscular Dystrophy treatment

Charity funds and drugmaker programs for Becker Muscular 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 Becker Muscular Dystrophy

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

The DMD gene page lists every condition Orphanet links to the gene and the open trials that name it.

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Trusted Becker Muscular Dystrophy resources

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

Active clinical trials for Becker Muscular Dystrophy

Use this Becker Muscular Dystrophy clinical trial finder to see the 13 studies recruiting patients and 5 not yet recruiting in the United States and worldwide, with eligibility criteria in plain English. Of these, 4 test a treatment and 14 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.

18 active trials worldwide
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Not yet recruitingObservational study, no treatment givenRecently updatedNCT07740057

Multimodal Study About Absence of Dp140 in Becker Muscular Dystrophy: Brain-muscle Imaging, Cognition and Muscle Function

Sponsor: Institut de Myologie, France

Monitoring patients with neuromuscular disorders is crucial but doesn't always allow for a sufficiently specific approach to all aspects of the condition. However, in the event of future treatment, it is essential for clinical and research teams to have as much information as pos...

Ages 18 Years to 50 Years1 location
Expected to start Dec 2026Updated 1 week agoEst. Dec 2029 (~3y 1m)
RecruitingObservational study, no treatment givenRecently updatedNCT07609394

Duchenne Electronic Health Record Study

Intervention: Observational study with patients who may be treated with various disease-modifying therapies

Sponsor: The Duchenne Registry

This study aims to collect retrospective and prospective, long-term data of patients with dystrophinopathy (including Duchenne, Becker, and female carriers) through electronic transfer. At select clinics across the United States, electronic health record (EHR) data from consented...

Ages not specified
Started Dec 2022Updated 1 week agoEst. Dec 2035 (~9y 1m)
RecruitingObservational study, no treatment givenRecently updatedNCT05019625

Biomarker Development for Muscular Dystrophies

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 5 Years+
Started Feb 2015Updated 2 weeks agoEst. Jun 2027 (~7 months)
RecruitingTreatment trialRecently updatedNCT06833489

Transcriptomic Analysis to Put an End to Misdiagnosis in Patients With Rare Muscle Diseases

Intervention: ARN extraction from muscle biopsies

Sponsor: Assistance Publique Hopitaux De Marseille

Since 2017, more than 250 analyses performed at the Molecular Genetics Laboratory of the Timone Enfant Hospital have yielded negative results in patients with rare genetic muscle diseases. The researchers hypothesise that some of these misdiagnosed patients carry pathogenic RNA (...

Ages not specified1 location
Started Jan 2025Updated 2 weeks agoEst. Dec 2026 (17 days)
RecruitingObservational study, no treatment givenRecently updatedNCT06147414

Development of Non-Invasive Prenatal Diagnosis for Single Gene Disorders

Intervention: Blood sample

Sponsor: Assistance Publique - Hôpitaux de Paris

Cell-free fetal DNA (cffDNA) is present in the maternal blood from the early first trimester of gestation and makes up 5%-20% of the total circulating cell-free DNA (cfDNA) in maternal plasma. Its presence in maternal plasma has allowed development of noninvasive prenatal diagnos...

Ages 18 Years+1 location
Started Oct 2024Updated 3 weeks agoEst. May 2027 (~6 months)
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Active trial locations21 cities in the US
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Trial Pipeline

Jan 2021 to Nov 2031
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Data from ClinicalTrials.gov, U.S. National Library of Medicine.
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Becker Muscular Dystrophy patient communities

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Companies developing Becker Muscular Dystrophy treatments

Drugmakers that list Becker Muscular Dystrophy in their rare disease portfolio, 4 in all.

Questions people ask about Becker Muscular Dystrophy