Muscular Dystrophy
Understanding the role of contraction-induced muscle injury
In muscular dystrophies like Becker and Duchenne, muscle contraction with everyday movements causes muscle injuries in the absence of fully functioning protective proteins. Over time, muscle fibers get replaced with fat and scar tissue. This disease process is called “contraction-induced muscle injury” and it is the root cause of functional challenges linked to serious muscle diseases like Becker and Duchenne, as well as other muscular dystrophies. Protecting muscle is a key goal in the management of muscular dystrophies.
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Becker Muscular Dystrophy Overview
Becker is a genetic, progressive neuromuscular disorder that imposes significant physical, emotional, financial and social impacts predominantly on males and their caregivers. Genetic mutations in the dystrophin gene resulting in Becker lead to contraction-induced muscle damage, which is the primary driver of muscle loss and impaired motor function in muscular dystrophies. Functional decline can begin at any age, and once that muscle loss begins, the decline in function is irreversible and continuous.
About Becker
A serious, inherited condition primarily affecting boys and men
Becker is genetic, meaning it begins at birth.
Muscle damage: a key disease hallmark
Contraction-induced muscle damage leads to irreversible muscle loss and loss of function, which can occur at any age.
No medicines approved specifically for Becker
Therapies are in development; multidisciplinary care is critical for optimal management.
Duchenne Muscular Dystrophy Overview
Duchenne is a severe, degenerative muscle disorder with a median life expectancy of around 30 years old. People living with Duchenne begin to lose their ability to walk without assistance by their early teens and nearly all will require the use of a wheelchair by the time they are in their mid-teens. Duchenne is the most common type of muscular dystrophy, and genetic mutations in the dystrophin gene result in contraction-induced muscle damage, which is the primary driver of irreversible muscle loss and impaired motor function.
About Duchenne
A serious, inherited condition affecting boys
Duchenne is genetic, meaning it begins at birth; primarily affects boys.
Muscle damage: a key disease hallmark
Contraction-induced muscle damage leads to irreversible muscle loss and loss of function; nearly all people with Duchenne will require a wheelchair in their mid-teens.
There is no cure for Duchenne
Therapies are available and in development; multidisciplinary care is critical for optimal management.
Sevasemten
Sevasemten is an orally administered first-in-class fast skeletal myosin inhibitor designed to preserve and protect unstable muscle against contraction-induced injury in individuals living with rare skeletal muscle disorders, including Becker and Duchenne.
Sevasemten is an investigational therapy that has not been approved for use in Becker muscular dystrophy or Duchenne muscular dystrophy by any regulatory agency, as its safety and effectiveness have not been established for the treatment of these diseases.
