This is a Study to Get More Information About Non Ambulatory Boys & Men With Duchenne Muscular Dystrophy
| Status: | Completed |
|---|---|
| Conditions: | Neurology, Orthopedic |
| Therapuetic Areas: | Neurology, Orthopedics / Podiatry |
| Healthy: | No |
| Age Range: | 7 - 22 |
| Updated: | 1/19/2017 |
| Start Date: | March 2010 |
| End Date: | November 2013 |
Clinical Outcomes Validation in Non Ambulatory and Young Boys/Men With Duchenne Muscular Dystrophy (DMD)
Many therapeutic trials in DMD exclude non-ambulatory boys and men. Rate of progression in
these non-ambulatory patients has been studied but consensus has not been reached for what
measures are most reliable and reproducible. Furthermore, any treatment trial would be
expected to demonstrate improved function and improvement in quality of life. Therefore,
function, strength, and quality of life must be understood and standardized. While the goal
of this proposal is to standardize clinical outcomes for therapeutic trials, careful
understanding of the progression of DMD in non ambulatory boys may also lead to better
medical treatment.
these non-ambulatory patients has been studied but consensus has not been reached for what
measures are most reliable and reproducible. Furthermore, any treatment trial would be
expected to demonstrate improved function and improvement in quality of life. Therefore,
function, strength, and quality of life must be understood and standardized. While the goal
of this proposal is to standardize clinical outcomes for therapeutic trials, careful
understanding of the progression of DMD in non ambulatory boys may also lead to better
medical treatment.
For Non-ambulatory study, boys must be unable to walk without assistive devices for greater
than or equal to one year.
Age at onset of study for non-ambulatory boys and men: greater than or equal to 7 years
through age 22 years.
Genetic or biopsy confirmation of dystrophinopathy or clinical diagnosis of DMD with
biopsy/genetic confirmation in a primary relative.
than or equal to one year.
Age at onset of study for non-ambulatory boys and men: greater than or equal to 7 years
through age 22 years.
Genetic or biopsy confirmation of dystrophinopathy or clinical diagnosis of DMD with
biopsy/genetic confirmation in a primary relative.
Inclusion Criteria:
1. For Non-ambulatory study, boys must be unable to walk without assistive devices for
greater than or equal to one year.
2. Age at onset of study for non-ambulatory boys and men: greater than or equal to 7
years through age 22 years.
3. Genetic or biopsy confirmation of dystrophinopathy or clinical diagnosis of DMD with
biopsy/genetic confirmation in a primary relative.
Exclusion Criteria:
1. For Non-ambulatory study, the inability to understand and cooperate with the testing
would exclude a subject. -
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