Duchenne Muscular Dystrophy Overview

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Learn About Duchenne Muscular Dystrophy

What is the definition of Duchenne Muscular Dystrophy?
Duchenne muscular dystrophy (DMD) affects the muscles, leading to muscle wasting that gets worse over time. DMD occurs primarily in males, though in rare cases may affect females. The symptoms of DMD include progressive weakness and loss (atrophy) of both skeletal and heart muscle. Early signs may include delayed ability to sit, stand, or walk and difficulties learning to speak. Muscle weakness is usually noticeable in early childhood. DMD is caused by genetic changes (DNA variants) in the DMD gene. DMD is inherited in an X-linked recessive pattern and may occur in people who do not have a family history of DMD. Diagnosis of DMD is based on the symptoms, clinical exam, and the result of a biopsy to remove a small piece of muscle for examination under a microscope. The result of genetic testing may also help confirm the diagnosis. Becker muscular dystrophy (BMD), a milder form of muscular dystrophy, is also caused by genetic changes in the DMD gene.
What are the alternative names for Duchenne Muscular Dystrophy?
  • Duchenne muscular dystrophy
  • DMD
  • Muscular dystrophy, Duchenne
  • Muscular dystrophy, pseudohypertrophic progressive, Duchenne type
Who are the top Duchenne Muscular Dystrophy Local Doctors?
Elite in Duchenne Muscular Dystrophy
Dr. Katherine D. Mathews
Neurology | Pediatrics | Pediatric Neurology
Elite in Duchenne Muscular Dystrophy
Dr. Katherine D. Mathews
Neurology | Pediatrics | Pediatric Neurology

State University Of Iowa

200 Hawkins Dr, 
Iowa City, IA 
319-356-1616
Languages Spoken:
English
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Accepting New Patients

Katherine Mathews is a Neurologist and a Pediatrics provider in Iowa City, Iowa. Dr. Mathews and is rated as an Elite provider by MediFind in the treatment of Duchenne Muscular Dystrophy. Her top areas of expertise are Limb-Girdle Muscular Dystrophy Type 2I, Limb-Girdle Muscular Dystrophy, Duchenne Muscular Dystrophy, and Friedreich Ataxia. Dr. Mathews is currently accepting new patients.

Zarife Sahenk
Elite in Duchenne Muscular Dystrophy
Dr. Zarife Sahenk
Pediatric Neurology | Neurology
Elite in Duchenne Muscular Dystrophy
Dr. Zarife Sahenk
Pediatric Neurology | Neurology

Nationwide Children's Hospital

700 Children's Drive, 
Columbus, OH 
614-722-2203
Languages Spoken:
English
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Zarife Sahenk is a Pediatric Neurologist and a Neurologist in Columbus, Ohio. Dr. Sahenk and is rated as an Elite provider by MediFind in the treatment of Duchenne Muscular Dystrophy. Their top areas of expertise are Duchenne Muscular Dystrophy, Becker Muscular Dystrophy, Charcot-Marie-Tooth Disease, and Limb-Girdle Muscular Dystrophy.

 
 
 
 
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Leanne M. Ward
Elite in Duchenne Muscular Dystrophy
Leanne M. Ward
Elite in Duchenne Muscular Dystrophy
Leanne M. Ward
401 Smyth Rd, R250H, K1H 8L1, 
Ottawa, ON, CA 

Leanne Ward practices in Ottawa, Canada. Ms. Ward and is rated as an Elite expert by MediFind in the treatment of Duchenne Muscular Dystrophy. Her top areas of expertise are Rickets, X-Linked Hypophosphatemia, Hypophosphatemia, and Malnutrition.

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What are the latest Duchenne Muscular Dystrophy Clinical Trials?
A Phase 3, Multinational, Randomized, Double-Blind, Placebo-Controlled Systemic Gene Transfer Therapy Study to Evaluate the Safety and Efficacy of SRP- 9001 in Non-Ambulatory and Ambulatory Subjects With Duchenne Muscular Dystrophy (ENVISION)
A Phase 3, Multinational, Randomized, Double-Blind, Placebo-Controlled Systemic Gene Transfer Therapy Study to Evaluate the Safety and Efficacy of SRP- 9001 in Non-Ambulatory and Ambulatory Subjects With Duchenne Muscular Dystrophy (ENVISION)
Enrollment Status: Recruiting
Publish Date: April 15, 2025
Intervention Type: Genetic
Study Phase: Phase 3

Summary: The study will evaluate the safety and efficacy of delandistrogene moxeparvovec gene transfer therapy in non-ambulatory and ambulatory males with DMD. This is a randomized, double-blind, placebo-controlled 2-part study. Participants will be in the study for approximately 128 weeks. All participants will have the opportunity to receive intravenous (IV) delandistrogene moxeparvovec in either Part 1 ...

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A Phase 1/2, Multicenter, Open-Label Study to Investigate the Safety, Tolerability, and Efficacy of a Single Intravenous Dose of SGT-003 in Ambulant Males With Duchenne Muscular Dystrophy (INSPIRE DUCHENNE)
A Phase 1/2, Multicenter, Open-Label Study to Investigate the Safety, Tolerability, and Efficacy of a Single Intravenous Dose of SGT-003 in Ambulant Males With Duchenne Muscular Dystrophy (INSPIRE DUCHENNE)
Enrollment Status: Recruiting
Publish Date: April 15, 2025
Intervention Type: Genetic
Study Phase: Phase 1/Phase 2

Summary: This is a multicenter, open-label, non-randomized study to investigate the safety, tolerability, and efficacy of a single intravenous (IV) infusion of SGT-003 in participants with Duchenne muscular dystrophy. There will be 2 cohorts in this study. Cohort 1 will include participants 4 to \<7 years of age. Cohort 2 will include participants 7 to \<12 years of age. All participants will receive SGT-0...

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Who are the sources who wrote this article ?

Published Date: May 02, 2022
Published By: Genetic and Rare Diseases Informnation Center