welcome to DuchenneXchange- a positively charged Duchenne muscular dystrophy community.
- join today!
This is a Study to Get More Information About Non Ambulatory Boys & Men With Duchenne Muscular Dystrophy
study id #: NCT01098708
condition: Duchenne Muscular Dystrophy
Many therapeutic trials in DMD exclude non-ambulatory boys and men. Rate of progression in these non-ambulatory patients has been studied but consensus has not been reached for what measures are most reliable and reproducible. Furthermore, any treatment trial would be expected to demonstrate improved function and improvement in quality of life. Therefore, function, strength, and quality of life must be understood and standardized. While the goal of this proposal is to standardize clinical outcomes for therapeutic trials, careful understanding of the progression of DMD in non ambulatory boys may also lead to better medical treatment.
mechanism of action: No pharmaceutical intervention
last updated: November 22, 2018
start date: March 2010
estimated completion: November 2013
size / enrollment: 50
study description: For Non-ambulatory study, boys must be unable to walk without assistive devices for greater than or equal to one year.
Age at onset of study for non-ambulatory boys and men: greater than or equal to 7 years through age 22 years.
Genetic or biopsy confirmation of dystrophinopathy or clinical diagnosis of DMD with biopsy/genetic confirmation in a primary relative.
- Dystrophin gene mutations that predispose to early onset cardiomyopathy [ Time Frame: 3 years ]
• For Non-ambulatory study, boys must be unable to walk without assistive devices for greater than or equal to one year.
• Age at onset of study for non-ambulatory boys and men: greater than or equal to 7 years through age 22 years.
• Genetic or biopsy confirmation of dystrophinopathy or clinical diagnosis of DMD with biopsy/genetic confirmation in a primary relative.
For Non-ambulatory study, the inability to understand and cooperate with the testing would exclude a subject.
Extension Study of BMN 044 in Duchenne Muscular Dystrophy (DMD)The aim of this study is to provide cont...
A Phase 1b Study of SMT C1100 in Subjects With Duchenne Muscular Dystrophy (DMD)The purpose of this study is to determin...
Effect of EPA and DHA in the Inflammation and Metabolic Disorders in DMD/DMB PatientsThe purpose of this study is to evaluate...
A Randomized, Double-blind, Placebo-controlled Study of SRP-9001 for Duchenne Muscular Dystrophy (DMD)The purpose of this study is to evaluate...
Stem Cell Therapy in Duchenne Muscular DystrophyThe purpose of this study was to study t...
Contemporary cardiac issues in Duchenne muscular dystrophyDuchenne muscular dystrophy (DMD) is an ...
ReveraGen Announces First Patient Enrollment in International Pivotal Trial of Vamorolone in Duchenne Muscular Dystr...ReveraGen BioPharma, Inc. today announce...
Sarepta Therapeutics Announces Recipients of Route 79, The Duchenne Scholarship ProgramCAMBRIDGE, Mass., Sept. 05, 2019 (GLOB...
Ataluren in patients with nonsense mutation Duchenne muscular dystrophy (ACT DMD): a multicentre, randomised, double...Background: Duchenne muscular dystrophy...
Evaluation of Hand Orthoses in Duchenne Muscular DystrophyPurpose: The purpose of this study was ...