welcome to DuchenneXchange- a positively charged Duchenne muscular dystrophy community.
- join today!
- log in
Duchenne Muscular Dystrophy Tissue Bank for Exon Skipping
study id #: NCT01772043
condition: Duchenne Muscular Dystrophy
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.
mechanism of action: No pharmaceutical intervention
last updated: November 22, 2018
start date: September 2012
estimated completion: August 2016
size / enrollment: 53
The purpose of this tissue bank is to collect blood and skin samples from participants who are diagnosed with Duchenne muscular dystrophy (DMD) and carry one of nine specific changes in the dystrophin gene. The specific dystrophin changes that we are interested in studying are those that would work with exon-skipping therapies in patients with DMD, specifically deletions of the follow exons: 10-52, 13-50, 29-50, 43-52, 44, 43-50, 45-50, 45-52, 46, 46-47, 46-48, 46-49, 46-51, 46-53, 46-55, 46-60, 47-50, 47-52, 48-50, 49-50, 50, 52, 52-63, 48-52, 49-52, 50-52.
These blood and skin samples will be held in a tissue bank at Carolinas Medical Center for future DMD research.
- Tissue Collection [ Time Frame: 1 day ]
Collection of blood, skin and optional muscle samples
• 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: Investigator assessment of inability to comply with blood and skin sample collection
Electrical Impedance Myography and Ultrasound as Biomarkers of Duchenne Muscular DystrophyResearchers at Children's Hospital Bosto...
Study of Ataluren for Previously Treated Patients With nmDBMD in Europe, Israel, Australia, and CanadaDuchenne/Becker muscular dystrophy (DBMD...
Plus Epicatechin Duchenne Muscular Dystrophy in Non-ambulatory AdolescentsThis single center open-label pilot stud...
Wave Life Sciences Duchenne Muscular Dystrophy Clinical Trial Selected for FDA Complex Innovative Trial Designs Pilo...Wave Life Sciences Ltd., a biotechnology...
Catabasis Quarterly: Updates on edasalonexent and clinical trialsWe are pleased to announce that we have ...
A Randomized, Double-blind, Placebo-controlled Study of SRP-9001 for Duchenne Muscular Dystrophy (DMD)The purpose of this study is to evaluate...
Pfizer doses first patient using investigational mini-dystrophin gene therapy for the treatment of Duchenne muscular...Pfizer Inc. has initiated a Phase 1b cli...
Cardiac and Skeletal Muscle Effects in the Randomized HOPE-Duchenne TrialObjective: To assess the feasibility, s...
Antisense Therapeutics to commence muscular dystrophy trialsAntisense Therapeutics is planning to un...