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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

Prevelance

1.7 - 2.7 / 100,000

3,310 - 29,790

US Estimated

5,135 - 46,215

Europe Estimated

Age of Onset

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ICD-10

G71.0

Inheritance

Autosomal dominant

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Autosomal recessive

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Mitochondrial/Multigenic

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X-linked dominant

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X-linked recessive

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5 Facts you should know

FACT

1

Primary signs of BMD are progressive weakness and wasting of the skeletal and cardiac muscles

FACT

2

It primarily affects males

FACT

3

Muscle weakness usually becomes apparent between the ages of 5 and 15

FACT

4

 In some cases, cardiomyopathy is the first sign

FACT

5

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

Becker muscular dystrophy (BMD) is also know as...

Becker muscular dystrophy (BMD)

Benign pseudohypertrophic muscular dystrophy; Becker's muscular dystrophy; Muscular dystrophy, Becker type;

What’s your Rare IQ?

With BMD, which part of the body often becomes enlarged?

Common signs & symptoms

Abnormal urinary color

Difficulty climbing stairs

Difficulty walking

Exercise intolerance

Decreased ability to exercise

Elevated serum creatine kinase

Elevated blood creatine phosphokinase
Increased CPK

Myalgia

Muscle ache

Myoglobinuria

Elevated hepatic transaminase

High liver enzymes

Current treatments

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.[3]

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.[6]

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.[7242]

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.[7242] There are a number of additional therapies for BMD being studied.[7242] 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).[6][7]

Top Clinical Trials

TitleDescriptionPhasesStatusInterventionsMore Information
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 1RecruitingDrug: EDG-5506Click here for more information
A Study to Assess Safety, Tolerability, and PK of EDG-5506 in Healthy Volunteers and Becker Muscular Dystrophy AdultThis 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 1Active, not recruitingDrug: EDG-5506
Drug: Placebo
Click here for more information
Safety and Biomarker Response to (+)-Epicatechin in Becker Muscular DystrophThe 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 1Active, not recruitingDrug: (+)-EpicatechinClick here for more information

Top Treatments in Research

AgentClass/Mechanism of ActionDevelopment StatusCompanyClinical StudiesMore Information
EDG-5506EDG-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 wastingPhase 1Edgewise TherapeuticsClick here for more informationClick here for more information
(+)-EpicatechinEpicatechin[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 1Epirium BioClick here for more informationClick here for more information