Edgewise Therapeutics 
(EDG-5506-210)

Study name: LYNX
A study to test safety, pharmacokinetics and biomarkers of muscle damage in boys with DMD ages 4-9.

An additional cohort, Cohort 2NS, to include participants (aged 4 to 7 years inclusive) not currently treated with corticosteroids, will enroll approximately 9 participants after Cohort 2 safety review and in parallel with the additional cohorts.

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  Current Status:
Active; Recruiting

Edgewise Therapeutics
(EDG-5506-201)

Study name: CANYON
A study for men with Becker muscular dystrophy ages 18-55.

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 Current Status:
Active; Recruiting

Edgewise Therapeutics
(EDG-5506-215)

Study name: FOX
A FOX study is a Phase 2, multicenter trial evaluating safety, pharmacokinetics, and biomarkers in children and adolescents (aged 6-17) with Duchenne muscular dystrophy previously treated with gene therapy. It includes a randomized, double-blind, placebo-controlled Part A, followed by an open-label Part B.

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 Current Status:
Active; Recruiting

Dyne Therapeutics
(Dyne-251-DMD-201)

Study name: Deliver
A study to evaluate safety, tolerability, and dystrophin levels in males with DMD ages 4 to 16 who have mutations amenable to exon 51 skipping therapy. The study will also measure muscle function, exon skipping, and pharmacokinetics.

Catalyst Pharmaceuticals, Inc. (DMD-001)

Registry Study to observe long-term safety of Vamorolone (AGAMREE®) in patients iwth Duchenne Muscular Dystrophy.

This is a multi-center, observational, prospective, longitudinal registry designed to collect data in male patients aged 2 years and older with DMD treated with AGAMREE®. 

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 Current Status:
Enrollment starting soon

Investigator Initiation Studies: 

Registry - Description

This study is intended to collect data from medical records on patients who have Duchenne Muscular Dystrophy

Overview:
This is a data collection study that will allow researchers to gather information to better understand Duchenne Muscular Dystrophy . Examples of data collected form medical records include descriptions of muscle function, lab results, dexascans, and outcomes in response to treatment.

What we're hoping for:
We are hoping that the information gathered by this study may be used to help improve identification and treatment of children with dystrophin disorders.

Biorepository

Description:
Seeking boys and men with Duchenne muscular dystrophy (DMD) or Becker Muscular Dystrophy (BMD) to provide samples including blood, plasma, serum, urine, as well as the remainders of tissue samples collected as part of their usual clinical care for a biorepository.

Overview:
We are collecting biological samples including an annual blood plasma and serum sample and a urine sample from volunteers with Duchenne muscular dystrophy (DMD) and Becker Muscular Dystrophy (BMD) to support future scientific investigation into these rare disorders.

What we're hoping for:
We hope to collect biological samples from boys and men with Duchenne muscular dystrophy (DMD) and Becker muscular dystrophy (BMD) to support future scientific investigation.

f-MRI

Seeking male participants aged 10-21 years of age who have been diagnosed with Duchenne Muscular Dystrophy (DMD) for participation in an imaging study.

Overview:
The purpose of this research is to learn how different mutations of Duchenne Muscular Dystrophy mutations present in a neuropsycholgical view.

What we're hoping for:
The purpose of this research is to learn how different mutations of Duchenne Muscular Dystrophy mutations are present in a neuropsychological view.