Home/Rare Diseases/Becker Muscular Dystrophy

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.

View 81 active trialsMatch me to a trial

About Becker 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.

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 in about 15% of patients), and rarely respiratory muscles. BMD shows slower progression than DMD, with some patients retaining ambulation into the third to fourth decade of life. 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.

Common Symptoms of Becker Muscular Dystrophy

Recognizing the signs of Becker Muscular Dystrophy early can lead to faster diagnosis and better outcomes. Symptoms may vary in severity from person to person. If you or a loved one are experiencing any of the following, consider speaking with a specialist.

  • 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 in some cases

Who Becker Muscular Dystrophy Affects

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.

Find Your Next Step

Answer a few questions and we'll point you to the right tools and information for where you are right now.

Where are you in your Becker Muscular Dystrophy journey?

Help Paying for Becker Muscular Dystrophy Treatment

Charity funds and drugmaker programs for Becker Muscular Dystrophy, checked at the source. Pick your insurance to see what fits.

Your insurance
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 September 28, 2026.

Open the full patient assistance finder →

Side Effect Explorer

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.

Loading side effect data...

Questions about side effects?
I can help you understand what these reports mean
Tap to start:
Or start with one of these

Genetic Testing

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.

Finding labs...

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 28 studies recruiting patients and 2 opening soon in the United States and worldwide, with eligibility criteria in plain English. These studies play a critical role in advancing care for neurological & neuromuscular conditions and may offer access to treatments not yet widely available. Each trial below is sourced directly from ClinicalTrials.gov, with eligibility criteria translated into plain English to help patients and caregivers evaluate whether a study may be a fit.

TrialsSite mapPipeline 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.

81 active trials worldwide
Filter:
Sort:
RECRUITINGPHASE3Recently updatedNCT07160634

A Study of SGT-003 Gene Therapy in Ambulant Males With Duchenne Muscular Dystrophy (IMPACT DUCHENNE)

Intervention: SGT-003, Placebo

Sponsor: Solid Biosciences Inc.

This is a Phase 3, double-blind, placebo-controlled study with the primary objective of evaluating the efficacy of a single IV infusion of SGT-003 in pediatric ambulant male participants with DMD. The secondary objectives include the evaluation of additional efficacy and safety o...

Ages 7 Years – 11 Years7 locations
Started Oct 2025Updated yesterdayEst. Jan 2029 (~2y 4m)
RECRUITINGRecently updatedNCT06564974

Registry Study to Observe Long-term Safety of Vamorolone (AGAMREE®) in Patients With Duchenne Muscular Dystrophy-SUMMIT

Intervention: Vamorolone

Sponsor: Catalyst Pharmaceuticals, Inc. · ICON plc + 2 more

The goal of this study is to collect additional information on the safety of long-term treatment with AGAMREE® and to explore long-term clinical impact of AGAMREE® on quality of life, as assessed by standardized patient-reported outcome measures (QoL questionnaires) in male patie...

Ages 2 Years+33 locations
Started Sep 2024Updated 5 days agoEst. Feb 2032 (~5y 5m)
RECRUITINGPHASE1Recently updatedNCT04626674

A Gene Transfer Therapy Study to Evaluate the Safety of and Expression From Delandistrogene Moxeparvovec (SRP-9001) in Participants With Duchenne Muscular Dystrophy (DMD) - Non-Ambulatory Cohort

Intervention: delandistrogene moxeparvovec

Sponsor: Sarepta Therapeutics, Inc. · Hoffmann-La Roche

Cohort 8 (non-ambulatory participants) is currently enrolling new participants. Enrollment for Cohorts 1 through 7 has been completed.

Ages 2 Years+12 locations
Started Nov 2020Updated 2 weeks agoEst. Dec 2027 (~1y 3m)
NOT YET RECRUITINGPHASE4Recently updatedNCT07542314

Study to Evaluate the Safety and Effectiveness of ELEVIDYS in Participants With Duchenne Muscular Dystrophy Treated in a Post-Marketing Setting

Intervention: ELEVIDYS, Sirolimus, Glucocorticoids, Antibiotics

Sponsor: Sarepta Therapeutics, Inc.

The primary objective of this study is to evaluate acute liver injury (ALI) rates associated with ELEVIDYS with the addition of sirolimus as an adjunct prophylactic immunosuppression agent.

Ages 4 Years+1 location
Started Aug 2026Updated 1 month agoEst. Aug 2027 (~11 months)
RECRUITINGPHASE1Recently updatedNCT07172971

Sodium/Glucose Cotransporter-2 Inhibitors (SGLT2i) Therapy in Duchenne Cardiomyopathy

Intervention: SGLT-2 inhibitor, SGLT2 inhibitor

Sponsor: Vanderbilt University Medical Center

This is a pharmacokinetic study (PK Study) to better understand empagliflozin dosing in pediatric Duchenne muscular dystrophy patients. Empagliflozin is currently used off-label in this population due to the mortality benefits seen in adult cardiomyopathy and heart failure. Inves...

Ages 8 Years – 18 Years1 location
Started Jul 2026Updated 1 month agoEst. Aug 2027 (~10 months)
Get trial alerts

Get notified when new Becker Muscular Dystrophy trials open or existing trials change status, add sites, or update eligibility.

We never share your email. Unsubscribe anytime.
Find Becker Muscular Dystrophy trials near you, ranked by distance →
Active trial locations55 cities in the US
+47 more

Trial Pipeline

Jan 2021 to Sep 2031
2021
2023
2025
2027
2029
2031
now
Phase 1
Phase 2
Phase 3
Phase 4
Observational
Observational
RecruitingOpening soonDelayed startTodayHover a bar for trial details
Need help understanding these trials?
Type your own question with a little about your situation, and get an answer with sources.
Tap to start:
Or start with one of these
Run a Becker Muscular Dystrophy foundation or patient group?
You can put this live trial list on your own website. It updates itself, and it's free.
Get the embed code →

Data from ClinicalTrials.gov, U.S. National Library of Medicine.
Always talk to your doctor before considering a clinical trial.

Patient Communities

Connect with other Becker Muscular Dystrophy patients, caregivers, and advocacy groups across Facebook groups, Reddit communities, and YouTube channels. These patient communities offer peer support, shared experiences, caregiver resources, and real-time discussion about Becker Muscular Dystrophy treatments, clinical trial participation, and day-to-day disease management.

Find the right community
Type your own question with a little about your situation, and get an answer with sources.
Tap to start:
Or start with one of these

Related Neurological & Neuromuscular Conditions

Other rare diseases in the neurological & neuromuscular category. Patients with Becker Muscular Dystrophy may find relevant research, shared treatment pathways, or overlapping clinical trials among these related conditions.

Companies Developing Becker Muscular Dystrophy Treatments

6 pharmaceutical companies have Becker Muscular Dystrophy in their rare disease portfolio

Frequently Asked Questions About Becker Muscular Dystrophy