Epidemiology, natural course and registration of dystrophinopathies in the Netherlands

Epidemiology, natural course and registration of dystrophinopathies in the Netherlands - Dystrophinopathies in the Netherlands

Status

Recruiting

Phases

Unknown

Study type

Observational

Source

NL-OMON

Registry ID

NL-OMON32292

Enrollment

800

Registered

2008-04-25

Start date

2008-06-01

Completion date

Unknown

Last updated

2024-05-13

For informational purposes only — not medical advice. Sourced from public registries and may not reflect the latest updates. Terms

Conditions

Duchenne/Becker muscular dystrophy

Interventions

None listed

Eligibility

Age

2 Years to 99 Years

Inclusion criteria

Inclusion criteria: Progressive muscle weakness (from childhood) Confirmation of dystrophinopathy diagnosis by a mutation in the dystrophin gene or the absence of dystrophin in a muscular biopt

Exclusion criteria

Exclusion criteria: no informed consent

Design outcomes

Primary

Measure	Time frame
- registration in national and international database - year of birth - age when able to walk independantly for the first time - age at diagnosis Duchenne/Becker - mutation in dystrophin gene - level of education - age at becoming wheelchair dependant - scoliosis and possible surgery - cardiomyopathy - age at which time home ventilation was initiated, if applicable - age at death - use of corticosteroids	—

Measure

Time frame

- registration in national and international database - year of birth - age when able to walk independantly for the first time - age at diagnosis Duchenne/Becker - mutation in dystrophin gene - level of education - age at becoming wheelchair dependant - scoliosis and possible surgery - cardiomyopathy - age at which time home ventilation was initiated, if applicable - age at death - use of corticosteroids

—

Secondary

Measure	Time frame
none	—

Countries

Netherlands

Outcome results