Background: Natural history data are essential for trial design in Duchenne (DMD) and Becker muscular dystrophy (BMD), but recruitment for observational studies can be challenging.Objective: We reviewed reasons why patients or caregivers declined participation, and compared characteristics of participants and non-participants to assess possible selection bias in four observational studies, three on DMD and one on BMD.Methods: Three pediatric DMD studies focused on cross-sectional cognitive function and brain MRI (DMDbrain, n=35 and DMDperfusion, n = 12), and on longitudinal upper extremity function and muscle MRI (DMDarm, n = 22). One adult BMD study assessed longitudinal functioning (n = 36). Considerations for non-participation were retro...
The number of clinical trials for Duchenne muscular dystrophy is increasing. Many trials require mus...
Objective: To understand the natural disease upper limb progression over 3 years of ambulatory and ...
We performed a 1-year longitudinal study of Six Minute Walk Test (6MWT), North Star Ambulatory Asses...
Contains fulltext : 225944.pdf (Publisher’s version ) (Open Access)BACKGROUND: Nat...
Background: Natural history data are essential for trial design in Duchenne (DMD) and Becker muscula...
BACKGROUND AND OBJECTIVES: Clinical trials of genotype-targeted treatments in Duchenne muscular dyst...
Dystrophinopathies are caused by mutations in DMD resulting in progressive muscle weakness. They are...
With the emergence of experimental therapies for Duchenne muscular dystrophy (DMD), it is fundamenta...
OBJECTIVE: To evaluate the suitability of real-world data (RWD) and natural history data (NHD) for u...
Objective: With the emergence of experimental therapies for Duchenne muscular dystrophy (DMD), it is...
Novel emerging therapies for Duchenne muscular dystrophy (DMD), such as antisense oligomer (AO) medi...
International audienceBACKGROUND: To explore clinical heterogeneity of Duchenne muscular dystrophy (...
BACKGROUND AND OBJECTIVES: Clinical trials of genotype-targeted treatments in Duchenne muscular dyst...
Background: Therapeutic trials are critical to improving outcomes for individuals diagnosed with Duc...
Objective:To correlate time to loss of ambulation (LoA) and different truncating DMD gene mutations ...
The number of clinical trials for Duchenne muscular dystrophy is increasing. Many trials require mus...
Objective: To understand the natural disease upper limb progression over 3 years of ambulatory and ...
We performed a 1-year longitudinal study of Six Minute Walk Test (6MWT), North Star Ambulatory Asses...
Contains fulltext : 225944.pdf (Publisher’s version ) (Open Access)BACKGROUND: Nat...
Background: Natural history data are essential for trial design in Duchenne (DMD) and Becker muscula...
BACKGROUND AND OBJECTIVES: Clinical trials of genotype-targeted treatments in Duchenne muscular dyst...
Dystrophinopathies are caused by mutations in DMD resulting in progressive muscle weakness. They are...
With the emergence of experimental therapies for Duchenne muscular dystrophy (DMD), it is fundamenta...
OBJECTIVE: To evaluate the suitability of real-world data (RWD) and natural history data (NHD) for u...
Objective: With the emergence of experimental therapies for Duchenne muscular dystrophy (DMD), it is...
Novel emerging therapies for Duchenne muscular dystrophy (DMD), such as antisense oligomer (AO) medi...
International audienceBACKGROUND: To explore clinical heterogeneity of Duchenne muscular dystrophy (...
BACKGROUND AND OBJECTIVES: Clinical trials of genotype-targeted treatments in Duchenne muscular dyst...
Background: Therapeutic trials are critical to improving outcomes for individuals diagnosed with Duc...
Objective:To correlate time to loss of ambulation (LoA) and different truncating DMD gene mutations ...
The number of clinical trials for Duchenne muscular dystrophy is increasing. Many trials require mus...
Objective: To understand the natural disease upper limb progression over 3 years of ambulatory and ...
We performed a 1-year longitudinal study of Six Minute Walk Test (6MWT), North Star Ambulatory Asses...