The natural history study of Becker muscular dystrophy

Recruiting

• Conditions: Becker muscular dystrophy / BMD; 10029317

• Registration Number: NL-OMON46870
• Lead Sponsor: eids Universitair Medisch Centrum

Brief Summary

Not available

Detailed Description

Not available

Study Timeline

• Last Update Posted: 23 April 2024

Recruitment & Eligibility

• Status: Recruiting
• Sex: Not specified
• Target Recruitment: 50

Inclusion Criteria

1. Diagnosis of BMD defined by:
-Male gender
-Progressive muscular weakness AND
-Elevated serum CPK-levels AND
-An in-frame mutation in the dystrophin gene AND/OR reduced amount of dystrophin protein in a muscle biopsy
2. BMD patients 18 years and older

Exclusion Criteria

Patients will be excluded from muscle biopsies if they use oral anticoagulants.

Study & Design

• Study Type: Observational invasive
• Study Design: Not specified

Primary Outcome Measures

Name	Time	Method
<p>1.To describe variability in clinical characteristics and natural history of<br /><br>BMD.<br /><br>-To describe the variability in and pattern of skeletal muscle involvement<br /><br>-To describe variability in functional impairment<br /><br>-To describe the variability in cardiac involvement<br /><br>-To describe whether pulmonary function is impaired<br /><br>-To describe cognitive functioning in BMD</p><br>