NL-OMON50307
Recruiting
Not Applicable
Outcome measures in Duchenne Muscular Dystrophy: A Natural History Study - Duchenne natural history study
Overview
- Phase
- Not Applicable
- Intervention
- Not specified
- Conditions
- Duchenne Muscular Dystrophy
- Sponsor
- eids Universitair Medisch Centrum
- Enrollment
- 20
- Status
- Recruiting
- Last Updated
- 2 years ago
Overview
Brief Summary
No summary available.
Investigators
Eligibility Criteria
Inclusion Criteria
- •NON AMBULANT DMD PATIENTS
- •1\. Children, teenagers and adults from the age of 5 with DMD, who have lost the
- •ability to walk 10 meters with no support
- •2\. The diagnosis of DMD must be documented by genetic testing.
- •3\. Patients should preferably have deletions amenable of skipping of exons 51
- •or 53 or 45 or 44 or 46 or 50 or 52\.
- •4\. Patients should be capable of sitting upright in a wheelchair for at least
- •5\. Patients should be stable from a respiratory point of view., AMBULANT DMD
- •1\. Ambulant children from 5 years old and teenagers with DMD, and potential
- •candidates for future genetic therapies with antisense oligomer (AO) exon
Exclusion Criteria
- •NON AMBULANT DMD PATIENTS:, 1\. Patients who are currently involved in
- •interventional clinical trials aimed at restoring dystrophin will be excluded,
- •as their data could not be used to establish natural history of the disease
- •(participation in a previous interventional clinical trial prior to 6 months
- •from being recruited in the study is not an exclusion criterion)
- •2\. Patients with severe intellectual impairment, who would be unable to
- •cooperate with examination
- •3\. Patients/families we anticipate may have emotional/ psychological problems
- •if recruited in into a natural history study
- •4\. Symptomatic cardiac failure
Outcomes
Primary Outcomes
Not specified
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