Duchenne Muscular Dystrophy Tissue Bank for Exon Skipping

Trial ID or NCT#

NCT01772043,25513,19502

Status

not recruiting iconNOT RECRUITING

Purpose

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.

Eligibility Criteria

Ages Eligible for Study: Older than 4 Years
Sexes Eligible for Study: Male
Accepts Healthy Volunteers: No
Inclusion Criteria:
  1. - 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.
Exclusion Criteria:
  1. - Investigator assessment of inability to comply with blood and skin sample collection

Contact us to find out if this trial is right for you.

Contact

Shirley Paulose
650-724-3792