Rare Neurology News
Becker Muscular Dystrophy (BMD)
Becker muscular dystrophy is an X-linked recessive inherited disorder characterized by slowly progressing muscle weakness of the legs and pelvis
Age of Onset
5 Facts you should know
Primary signs of BMD are progressive weakness and wasting of the skeletal and cardiac muscles
It primarily affects males
Muscle weakness usually becomes apparent between the ages of 5 and 15
In some cases, cardiomyopathy is the first sign
Becker muscular dystrophy is related to Duchenne muscular dystrophy in that both result from a mutation in the dystrophin gene, but has a milder course
Interest over time
Common signs & symptoms
Abnormal urinary color
Difficulty climbing stairs
Decreased ability to exercise
Elevated serum creatine kinase
Elevated blood creatine phosphokinase
Elevated hepatic transaminase
High liver enzymes
There is currently no cure for Becker muscular dystrophy (BMD), and management aims to help with symptoms and improve the quality of life. Affected people are encouraged to remain active, because inactivity (such as bed rest) can make the muscle disease worse.
Physical therapy can help with stretching tight muscles and using assistive devices; occupational therapy can help with daily living skills; and speech therapy may help those with dysphagia (difficulty swallowing). Surgery may be needed for progressive scoliosis and development of contractures.
People with BMD should be monitored for orthopedic complications. Cardiac (heart) evaluations are recommended beginning at around 10 years old, or when symptoms first begin. Evaluations should be repeated at least every two years.
Some studies have shown that certain corticosteroids (such as prednisone or prednisolone) can slow the decline of muscle strength in people with Duchenne muscular dystophy; however, information about their use in people with BMD is limited. There are a number of additional therapies for BMD being studied. Potential future treatments for BMD may include gene therapy, exon skipping, ataluren, creatine, deacetylase inhibitors, myostatin inactivation, and cell therapy (myoblast treatment, and/or the use of stem cells).
Top Clinical Trials
|A Study of EDG-5506 in Adult Males With Becker Muscular Dystrophy (ARCH)||The ARCH study is an open-label, single-center, Phase 1b study of EDG-5506 to assess the safety and pharmacokinetics (PK) of EDG-5506 in adults with Becker muscular dystrophy (BMD).|
EDG-5506 is an investigational product intended to protect and improve function of dystrophic muscle fibers.
|Phase 1||Recruiting||Drug: EDG-5506||More Info|
|A Study to Assess Safety, Tolerability, and PK of EDG-5506 in Healthy Volunteers and Becker Muscular Dystrophy Adult||This Phase 1 study of EDG-5506 will assess the safety, tolerability, and pharmacokinetics (PK) and of EDG-5506 in adult healthy volunteers and in adults with Becker muscular dystrophy (BMD).||Phase 1||Active, not recruiting||Drug: EDG-5506|
|Safety and Biomarker Response to (+)-Epicatechin in Becker Muscular Dystroph||The safety and tolerability of three escalating doses of (+)-epicatechin will be assessed and early effectiveness measured by changes in plasma biomarkers, tissue biomarkers from muscle biopsies, cardiac imaging, and on clinical function assessments of participants' muscle strength.||Phase 1||Active, not recruiting||Drug: (+)-Epicatechin||More Info|
Top Treatments in Research
|Agent||Class/Mechanism of Action||Development Status||Company||Clinical Studies||More Information|
|EDG-5506||EDG-5506 is a small orally available molecule that targets the underlying cause of muscle dystrophy by halting the use-driven muscle fiber damage and scar tissue buildup that lead to muscle weakness and wasting||Phase 1||Edgewise Therapeutics||More Info||More Info|
|(+)-Epicatechin||Epicatechin[i] is a naturally-occurring substance (technically, a “monomeric flavanol”) found in a variety of common foods, including certain fruits, green and white tea, and, especially, cocoa.||Phase 1||Epirium Bio||More Info||More Info|