Duchenne Muscular Dystrophy Tissue Bank for Exon Skipping
Trial ID or NCT#
We will utilize the Cooperative International Neuromuscular Research Group (CINRG) network to collect and store tissue and blood from patients with Duchenne muscular dystrophy (DMD) with specific genetic mutations within the dystrophin gene that could be treated by antisense oligonucleotide (AO) drugs.
- - Age 4 and above
- - Diagnosis of DMD with a confirmed out-of-frame dystrophin gene deletions that could be corrected by skipping exon 45, 51, or 53 based on past genetic testing.
- - Investigator assessment of inability to comply with blood and skin sample collection
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About this Clinical Trial
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