welcome to DuchenneXchange- a positively charged Duchenne muscular dystrophy community.
- join today!
A Registered Cohort Study on Duchenne Muscular Dystrophy
study id #: NCT04012671
condition: Duchenne Muscular Dystrophy
Dystrophinopathy is a term of X-linked recessive genetic disease, including Duchenne Muscular Dystrophy, Becker Muscular Dystrophy, and the X-linked dilated cardiomyopathy. The aim of this study is to determine the clinical spectrum and natural progression of dystrophinopathy in a prospective multicenter natural history study, to assess the clinical, genetic of patients with dystrophinopathy to optimize clinical management.
mechanism of action: No pharmaceutical intervention
last updated: December 04, 2019
start date: July 1, 2019
estimated completion: December 31, 2039
phase of development: N/A
size / enrollment: 2000
- Age at death [ Time Frame: 20 years ]
The time when patient die
• Eligible Sexes: all
• Beyond 2 years old
• Diagnosis with Duchenne Muscular Dystrophy, and female carriers, genotypically confirmed
• Diagnosis should be supported by muscle biopsy, if no genetic confirmation.
• Presence of other clinically significant illness
other Duchenne content
Cuixia Tian, MDDr. Cuixia Tian is a neuromuscular disea...
Efficacy, Safety, and Tolerability Rollover Study of Eteplirsen in Subjects With Duchenne Muscular DystrophyThe primary objective of this study is t...
Digital Droplet PCR for the Absolute Quantification of Exon Skipping Induced by Antisense Oligonucleotides in (Pre-)...Antisense oligonucleotides (AONs) in cli...
A Study to Evaluate Safety, Tolerability, and Efficacy of Eteplirsen in Patients With Duchenne Muscular DystrophyThe purpose of this extension study is t...
PTC Therapeutics Announces Initial Data from Patient Registry Demonstrating Translarna™ (ataluren) Slows Disease P...PTC Therapeutics, Inc. today announced p...
An Extension Study to Assess Vamorolone in Boys With Duchenne Muscular Dystrophy (DMD)The main purposes of this study are to s...
Gene Therapy OverviewIn this brief document, we want to expla...
Farida Abid, MDDr. Farida Abid currently serves as a ne...