welcome to DuchenneXchange
- a positively charged Duchenne muscular dystrophy community.- join today!
- log in
A Prospective Natural History Study of Progression of Subjects With Duchenne Muscular Dystrophy
study id #: NCT01753804
condition: Duchenne Muscular Dystrophy
status: terminated
purpose:To characterize the natural history and progression of Duchenne Muscular Dystrophy (DMD) to help inform the design of future studies, to capture biomarkers of safety and disease progression and to provide comparative data for the development of rare exons for which formal controlled trials are not feasible.
intervention: Observational study
mechanism of action: No pharmaceutical intervention
results: https://clinicaltrials.gov/ct2/show/results/NCT01753804
last updated: November 22, 2018
rareRelated
-
Myocardial Involvement in Carriers of Duchenne Muscular Dystrophy: An MRI-studyCarriers of Duchenne muscular dystrophy ...
-
What are the TREAT-NMD Global Registries?New therapeutic strategies for neuromusc...
-
Lower Limb Flexibility in Duchenne Muscular Dystrophy: Effects on Functional PerformanceThe investigator investigated the effect...
-
Phase 1 Study of Edasalonexent (CAT-1004), an Oral NF-κB Inhibitor, in Pediatric Patients with Duchenne Muscular Dy...Background: Edasalonexent is an orally ...
-
Stacking Exercises Aid the Decline in FVC and Sick TimeDuchenne Muscular Dystrophy is complicat...
-
The Duchenne RegistryThe Duchenne Registry is an online, pati...
-
Jerry Mendell, M.D., Presented Positive Updated Results from the Four Children Dosed in the Gene Therapy Micro-dystr...Sarepta Therapeutics, Inc., a commercial...
-
For Many Boys With Duchenne Muscular Dystrophy, Bright Hope Lies Just Beyond ReachLucas was 5 before his parents, Bill and...
-
Ataluren in patients with nonsense mutation Duchenne muscular dystrophy (ACT DMD): a multicentre, randomised, double...Background: Duchenne muscular dystrophy...