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Duchenne Muscular Dystrophy

Duchenne muscular dystrophy or DMD is the most common of the muscular dystrophies, affecting approximately 1 in every 3,500 newborn boys. It is caused by a fault in a gene called the dystrophin or DMD gene. A fault in this gene stops the body making a protein called dystrophin. This protein is important in muscle fibres, and its absence results in muscle weakness that gets worse over time because muscle cells break down and are gradually lost.

Because the dystrophin gene is on the X chromosome, Duchenne muscular dystrophy affects only boys. Girls have two X chromosomes, so if one of these is unaffected it can usually compensate for the faulty one, while boys have one X and one Y chromosome, so if their single copy of the dystrophin gene is faulty, they have the symptoms of DMD, while girls with one affected gene and one normal one usually won’t show symptoms but can be “carriers”. This means that the disease can be passed on in families – a mother who is a carrier has a 50:50 chance of having a son who is affected. But in up to about a third of cases, the mutation arises spontaneously in the boy.

Contents:

2018 Family Guides

2018 DMD Family Care Guide in Finnish 2018 DMD Family Care Guide in Hindi 2018 DMD Family Care Guide in Macedonian 2018 DMD Family Care Guide in Norwegian 2018 DMD Family Care Guide in Punjabi 2018 DMD Family Guide in German 2018 DMD Family Guide in Portuguese (Brazil) 2018 DMD Guide in Turkish Czech – DMD DMD Family Care Guide – Lebanese Arabic DMD Family Care Guide – Romanian DMD Family Care Guide in Arabic DMD Family Care Guide in Korean English UK – DMD English USA – DMD French DMD Family Care Guide 2018 Hebrew DMD Family Care Guide 2018 Russian – DMD Spanish DMD Family Guide 2018 Ukrainian – DMD

2010 Family Guides

Arabic – DMD Arabic – Israeli – DMD Bulgarian – DMD Chinese Simplified – DMD Chinese Traditional – DMD Danish – DMD Dutch – DMD English Canada – DMD Farsi – DMD Finnish – DMD French Canada – DMD Greek – DMD Hebrew – DMD Hungarian – DMD Italian – DMD Japanese – DMD Kyrgyz – DMD Latvian – DMD Lithuanian – DMD Norwegian – DMD Polish – DMD Portuguese – DMD Romanian – DMD Serbian – DMD Slovak – DMD Swedish – DMD Turkish – DMD Vietnamese – DMD

Patient organisations

Action Duchenne – United Kingdom Action Duchenne Aktion Benni & Co / Parent Project – Germany Aktion Benni & Co / Parent Project Association Française contre les Myopathies – AFM – France Association Française contre les Myopathies - AFM Canadian Neuromuscular Disease Registry – CNDR – Canada Canadian Neuromuscular Disease Registry - CNDR Charley's Fund – United States of America Charley's Fund CureDuchenne – United States of America CureDuchenne DMD Pathfinders – United Kingdom DMD Pathfinders DMD Serbia – Serbia DMD Serbia Duchenne Family Support Group – United Kingdom Duchenne Family Support Group Duchenne Ireland – Ireland Duchenne Ireland Duchenne Parent Project – Netherlands Duchenne Parent Project Duchenne Parent Project Spain – Spain Duchenne Parent Project Spain Duchenne Research Fund – United Kingdom Duchenne Research Fund Duchenne UK – United Kingdom Duchenne UK End Duchenne – Czech Republic End Duchenne Federacion Española de Enfermedades Neuromusculares (Federación ASEM) – Spain Federacion Española de Enfermedades Neuromusculares (Federación ASEM) Foundation to Eradicate Duchenne – United States of America Foundation to Eradicate Duchenne Fundation Gyógyító Jószándék – Hungary Fundation Gyógyító Jószándék InsamlingsStiftelsen för Muskel DystrofiForsknings – Sweden InsamlingsStiftelsen för Muskel DystrofiForsknings Joining Jack – United Kingdom Joining Jack Kolpingova rodina Smečno – Czech Republic Kolpingova rodina Smečno Little Steps association – Israel Little Steps association Muscular Dystrophy Association – United States of America Muscular Dystrophy Association Muscular Dystrophy UK – United Kingdom Muscular Dystrophy UK Myo-Care National Foundation – Egypt Myo-Care National Foundation PARENT PROJECT, zs – Czech Republic PARENT PROJECT, zs Parent Project – Genitori contro la Distrofia Musculare di Duchenne & Becker ONLUS Italy – Italy Parent Project - Genitori contro la Distrofia Musculare di Duchenne & Becker ONLUS Italy Parent Project Association – Romania Parent Project Association Parent Project France & Monaco – France Parent Project France & Monaco Parent Project Muscular Dystrophy – United States of America Parent Project Muscular Dystrophy Progena Swiss Duchenne Foundation – Switzerland Progena Swiss Duchenne Foundation Prothelia Inc – United States of America Prothelia Inc The Akari Foundation – United States of America The Akari Foundation The Gyógyító Jószándék Alapítvány – Healing Goodwill Foundation – Hungary United Duchenne Parent Project – Netherlands United Duchenne Parent Project University Clinical Centre, Medical University of Gdansk – Poland UPA! cura Duchenne – Mexico UPA! cura Duchenne

Meetings and events

Event: Duchenne Care Conference 2023

Start Date: June 7, 2023

Location: Online

Duchenne Care Conference 2023

Event: Duchenne Patient Academy 2022

Start Date: December 1, 2022

Location: Online

Duchenne Patient Academy 2022

Event: 3rd International FAIR Data ‘Visiting’ For Duchenne & Other Rare Diseases

Start Date: November 22, 2022

Location: online

3rd International FAIR Data ‘Visiting’ For Duchenne & Other Rare Diseases

Event: 3rd Symposium on Muscle-Bone interaction in Duchenne Muscular Dystrophy

Start Date: November 7, 2022

Location: Online

3rd Symposium on Muscle-Bone interaction in Duchenne Muscular Dystrophy

Event: World Duchenne Awareness Day 2022 Official Event

Start Date: September 7, 2022

Location:

World Duchenne Awareness Day 2022 Official Event

The aim of this overview is to inform patients and parents about the different therapeutic approaches for Duchenne muscular dystrophy currently under investigation, to describe the advantages and disadvantages of each approach and to list the hurdles that have to be overcome before these approaches can be applied to patients.

 

Find out more about what is currently happening in research >>

To find out more please click here.

The currently used mammalian models for DMD include several mice strains, two dog models and one cat model. The characteristics of each phenotype and pathology have been analyzed and compared in detail.

Find out more about DMD animal models

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