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Lead
Cooperative International Neuromuscular Research Group
We will utilize the Cooperative International Neuromuscular Research group (CINRG) network to collect cohorts of Becker muscular dystrophy (BMD) patients with in-frame deletions in the dystrophin gene. We will collect clinical data across multiple body systems and correlate these findings to the high-resolution deletion break-point mapping performed from the tissue samples. We will investigate the observed variability to deepen our understanding of molecular mechanisms relevant to the optimization of exon skipping therapeutic approaches.
Age
4–any
Sex
MALE
Healthy volunteers
Not accepted
You may be eligible if
Male
Age 4 or older
Diagnosis of BMD with an in-frame deletion in the dystrophin gene, where the boundaries of the mutations are confirmed.
You may not be if
Investigator assessment of inability to comply with protocol
