Duchenne muscular dystrophy (DMD) is a progressive, disabling genetic neuromuscular disorder in children caused by an absence of dystrophin protein, that helps maintain muscle integrity. It is inherited in an X-linked recessive manner. Duchenne muscular dystrophy (DMD) was first identified by the French neurologist Guillaume Benjamin Amand Duchenne in the 1860s. However in 1987, the protein linked to this gene was discovered and named dystrophin.The estimated incidence in children is 1 in 3,800. The absence of dystrophin in muscle cells makes them vulnerable and prone to damage. The initial symptoms include trouble climbing stairs, a waddling gait, and frequent falls, which typically appear in patients around the ages of 2 to 3. Muscle weakness is the primary symptom of Duchenne muscular dystrophy (DMD). Initially, the weakness affects the proximal muscles, followed by the distal limb muscles, leading to wheelchair dependency and severe complications in their late teens. Typically, the lower extremity muscles are impacted before the upper extremity muscles, children may struggle with activities such as jumping, running, and walking. Later on the heart and respiratory muscles are also affected, resulting in impaired cardiac and pulmonary function. Another type related to DMD is Becker muscular dystrophy (BMD), which is a milder form of the disease caused by deletions in the dystrophin gene. This results in comparatively mild muscle weakness, allowing individuals to maintain mobility throughout their lives, although it can also lead to severe muscle dysfunction similar to that seen in Duchenne muscular dystrophy (DMD). As the disease progresses, maintaining upper extremity function becomes crucial for preserving the ability to independently perform daily activities, such as self-care, using a computer, and operating manual or powered wheelchairs. Therefore, clinical assessments of disease-modifying treatments should prioritize the preservation of pulmonary, cardiac, and upper limb function to enhance quality of life.

Commonly used outcomes to evaluate Duchenne muscular dystrophy (DMD) include the Gowers’ maneuver, the time taken to get up from the floor, the 6-Minute Walk Test, increased tendency to fall compared to peers, and speech difficulties, all of which reflect the progression of the disease. In children with Duchenne muscular dystrophy (DMD), a multidisciplinary approach is employed to provide supportive therapies, such as treadmill walking, which helps enhance adaptive mechanisms necessary for coordination, body orientation, balance control, and maintaining an upright position. Additionally, cycling is considered an aerobic exercise that improves repetitive functional activities.

In patients with Duchenne muscular dystrophy (DMD), the upper limb maintain their strength and function longer than any other muscle group, unlike the respiratory system. Notably, the distal muscles of the upper limb tend to preserve their function for an extended period, even in the later stages of the disease to improve quality of life. Low-intensity aerobic exercises aimed at enhancing activity levels in children with Duchenne muscular dystrophy (DMD), such as assisted bicycle training, provide valuable practices that promote effective management and boost confidence, ultimately leading to an improved quality of life.The quality of life and life expectancy of patients with Duchenne muscular dystrophy (DMD) have improved due to advancements in technology and multidisciplinary care. Proposed treatments have effectively slowed disease progression, delaying both functional dependence and cardio respiratory impairments.

Finding optimal treatment for Duchenne muscular dystrophy has become challenging due to the complexity of the condition. The treatment options for Duchenne muscular dystrophy vary based on the child’s symptoms and age, with the goal of improving quality of life and postural control. While there are various tools available to assess upper limb function, postural control, and quality of life, these assessments are particularly important at stages when patients experience a loss of postural control and a decline in quality of life. The Alexander technique is used to enhance quality of life and postural tone, while also helping to modify body schema.This approach emphasizes the mind-body interaction, consciously inhibiting inefficient movements to enable the body to generate stronger and more comfortable movements, that allows the head and shoulder to balance forward and upward, thereby improving postural control of the upper limbs. Given that postural control and improved quality of life are benefits of the Alexander technique, it is based on the AID method. Here, A stands for awareness of muscle tone and poor postural habits, I represents the conscious inhibition of ineffective movements, and D refers to direction.

The Alexander technique is recognized as an effective treatment for upper limb postural control and quality of life; however, its specific effects on Duchenne muscular dystrophy (DMD) remain no known. So there should a study on effect of the Alexander technique on upper limb postural control and quality of life in children with DMD. Therefore the aim of this to determine the effects of the Alexander technique on upper limb posture control and quality of life in children with Duchenne muscular dystrophy

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A Gene Transfer Therapy Study to Evaluate the Safety of and Expression From Delandistrogene Moxeparvovec (SRP-9001) in Participants With Duchenne Muscular Dystrophy (DMD)

Actively Recruiting
2+ Years
Restoring or Replacing Dystrophin
Actively Recruiting
Therapeutic Approach: Restoring or Replacing Dystrophin
Variant Requirement: Duchenne - variant criteria varies by cohort
Industry/Sponsor: Sarepta Therapeutics, Inc.
Ambulation: Varies by cohort
Steroid Use: Varies by cohort
Eligible Sexes: Male
Clinical Trial NCT ID: NCT04626674
Location: United States
Next Steps:

A Study Evaluating the Real-World Experience of Givinostat in Patients With Duchenne Muscular Dystrophy

Actively Recruiting
6+ Years
Observational
Actively Recruiting
Therapeutic Approach: Observational
Variant Requirement: Duchenne - No specific requirement
Industry/Sponsor: ITF Therapeutics LLC
Ambulation: No specific requirement
Steroid Use: No specific requirement
Eligible Sexes: All
Clinical Trial NCT ID: NCT07127978
Location: United States
Next Steps:

A Study of SGT-003 Gene Therapy in Duchenne Muscular Dystrophy (INSPIRE DUCHENNE)

Actively Recruiting
0 Years to <18 Years
Restoring or Replacing Dystrophin
Actively Recruiting
Therapeutic Approach: Restoring or Replacing Dystrophin
Variant Requirement: Duchenne - Any deletion in exons 1 to 11 or 42 to 45 are excluded
Industry/Sponsor: Solid Biosciences Inc.
Ambulation: Varies by cohort
Steroid Use: Cohorts 1,2,4,&5 - Stable course of steroids; Cohort 3 - N/A
Eligible Sexes: Male
Clinical Trial NCT ID: NCT06138639
Next Steps:

A Study to Investigate the Safety and Biodistribution of a Single Intrathecal (IT) Injection of INS1201 in Ambulatory Males With Duchenne Muscular Dystrophy (DMD)

Actively Recruiting
2 Years to <5 Years
Restoring or Replacing Dystrophin
Actively Recruiting
Therapeutic Approach: Restoring or Replacing Dystrophin
Variant Requirement: Duchenne - variants in exons 18 to 58
Industry/Sponsor: Insmed Gene Therapy LLC
Ambulation: Ambulatory
Steroid Use: No specific requirement
Eligible Sexes: Male
Clinical Trial NCT ID: NCT06817382
Location: United States
Next Steps:

AFFINITY BEYOND: Anti-AAV8 Antibody Assessment Study of Males With DMD

Actively Recruiting
0 Years to <25 Years
Observational
Actively Recruiting
Therapeutic Approach: Observational
Variant Requirement: Duchenne - No specific requirement
Industry/Sponsor: REGENXBIO Inc.
Ambulation: No specific requirement
Steroid Use: No specific requirement
Eligible Sexes: Male
Clinical Trial NCT ID: NCT05683379
Location: United States
Next Steps:

AFFINITY DUCHENNE: RGX-202 Gene Therapy in Participants With Duchenne Muscular Dystrophy (DMD)

Actively Recruiting
1 to <12 years
Restoring or Replacing Dystrophin
Actively Recruiting
Therapeutic Approach: Restoring or Replacing Dystrophin
Variant Requirement: Duchenne - Part 1: Excluding variants in exons 1-17, Part 2 and 3: Excluding deletions or point mutations in exons 8, 9, and/or 10
Industry/Sponsor: REGENXBIO Inc.
Ambulation: Ambulatory
Steroid Use: Varies by cohort and age
Eligible Sexes: Male
Clinical Trial NCT ID: NCT05693142
Next Steps:

An Observational Study Comparing Delandistrogene Moxeparvovec With Standard of Care in Participants With Duchenne Muscular Dystrophy

Actively Recruiting
4+ Years
Restoring or Replacing Dystrophin
Actively Recruiting
Therapeutic Approach: Restoring or Replacing Dystrophin
Variant Requirement: Duchenne - Excluding any deletion of exon 8 and/or 9
Industry/Sponsor: Sarepta Therapeutics, Inc.
Ambulation: No specific requirement
Steroid Use: Stable course of steroids
Eligible Sexes: Male
Clinical Trial NCT ID: NCT06270719
Location: United States
Next Steps:

DMD Voice: Qualitative Interviews With Patients and Caregivers

Actively Recruiting
10+ Years
Observational
Actively Recruiting
Therapeutic Approach: Observational
Variant Requirement: Duchenne - No specific requirement
Industry/Sponsor: Red Nucleus Enterprise Solutions, LLC
Ambulation: No specific requirement
Steroid Use: No specific requirement
Eligible Sexes: Male
Clinical Trial NCT ID: NCT06925269
Location: United States
Next Steps:

Establishing Walking-related Digital Biomarkers in Rare Childhood Onset Progressive Neuromuscular Disorders

Actively Recruiting
5+ Years
Observational
Actively Recruiting
Therapeutic Approach: Observational
Variant Requirement: Duchenne - No specific requirement
Industry/Sponsor: Columbia University
Ambulation: Ambulatory
Steroid Use: Stable course of steroids
Eligible Sexes: All
Clinical Trial NCT ID: NCT06839469
Location: United States
Next Steps:

Givinostat in Duchenne's Muscular Dystrophy Long-term Safety and Tolerability Study

Actively Recruiting
6+ Years
Improving Muscle Growth & Protection
Actively Recruiting
Therapeutic Approach: Improving Muscle Growth & Protection
Variant Requirement: Duchenne - No specific requirement
Industry/Sponsor: Italfarmaco
Ambulation: Ambulatory or non-ambulatory
Steroid Use: No specific requirement
Eligible Sexes: Male
Clinical Trial NCT ID: NCT03373968
Next Steps:

Magnetic Resonance Imaging and Biomarkers for Muscular Dystrophy

Actively Recruiting
5 Years to <63 Years
Observational
Actively Recruiting
Therapeutic Approach: Observational
Variant Requirement: No specific requirement
Industry/Sponsor: University of Florida
Ambulation: Varies by cohort
Steroid Use: No specific requirement
Eligible Sexes: Male
Clinical Trial NCT ID: NCT01484678
Location: United States
Next Steps:

Modulation of SERCA2a of Intra-Myocytic Calcium Trafficking in Cardiomyopathy Secondary to Duchenne Muscular Dystrophy

Actively Recruiting
18+ Years
Regulating Calcium Balance
Actively Recruiting
Therapeutic Approach: Regulating Calcium Balance
Variant Requirement: Duchenne - No specific requirement
Industry/Sponsor: Sardocor Corp.
Ambulation: No specific requirement
Steroid Use: Stable course of steroids
Eligible Sexes: Male
Clinical Trial NCT ID: NCT06224660
Location: United States
Next Steps:

Molecular Analysis of Patients With Neuromuscular Disease

Actively Recruiting
All ages
Observational
Actively Recruiting
Therapeutic Approach: Observational
Variant Requirement: Diagnosis of a neuromuscular disorder for self or family member
Industry/Sponsor: Boston Children's Hospital
Ambulation: No specific requirement
Steroid Use: No specific requirement
Eligible Sexes: All
Clinical Trial NCT ID: NCT00390104
Location: United States
Next Steps:

NS-089/NCNP-02-201 in Boys With Duchenne Muscular Dystrophy (DMD)

Actively Recruiting
4 Years to <15 Years
Restoring or Replacing Dystrophin
Actively Recruiting
Therapeutic Approach: Restoring or Replacing Dystrophin
Variant Requirement: Duchenne - Amenable to exon 44 skipping
Industry/Sponsor: NS Pharma, Inc.
Ambulation: Ambulatory
Steroid Use: Stable course of steroids
Eligible Sexes: Male
Clinical Trial NCT ID: NCT05996003
Next Steps:

Once Weekly Infant Corticosteroid Trial for DMD

Actively Recruiting
1 Month to 30 Months
Reducing Inflammation
Actively Recruiting
Therapeutic Approach: Reducing Inflammation
Variant Requirement: Duchenne - No specific requirement
Industry/Sponsor: Nationwide Children's Hospital
Ambulation: No specific requirement
Steroid Use: Steroid-naive
Eligible Sexes: Male
Clinical Trial NCT ID: NCT05412394
Location: United States

Open-label Study of WVE-N531 in Patients With Duchenne Muscular Dystrophy (FORWARD-53)

Actively Recruiting
4 Years to <19 Years
Restoring or Replacing Dystrophin
Actively Recruiting
Therapeutic Approach: Restoring or Replacing Dystrophin
Variant Requirement: Duchenne - Amenable to exon 53 skipping
Industry/Sponsor: Wave Life Sciences Ltd.
Ambulation: Part A/B - Either; Part C - Ambulatory
Steroid Use: Stable course of steroids
Eligible Sexes: Male
Clinical Trial NCT ID: NCT04906460
Next Steps:

PBGENE-DMD Phase 1/2a Safety and Preliminary Efficacy Study in Duchenne Muscular Dystrophy (FUNCTION-DMD)

Actively Recruiting
2 Years to <8 Years
Restoring or Replacing Dystrophin
Actively Recruiting
Therapeutic Approach: Restoring or Replacing Dystrophin
Variant Requirement: Duchenne - variant fully contained between exons 45-55
Industry/Sponsor: Precision BioSciences, Inc.
Ambulation: Ambulatory
Steroid Use: No specific requirement
Eligible Sexes: Male
Clinical Trial NCT ID: NCT07429240
Location: United States
Next Steps:

Phase 2, Randomized, Placebo-controlled Proof-of-concept Study of SAT-3247 in Pediatric Ambulatory Patients (BASECAMP)

Actively Recruiting
7 Years to <10 Years
Improving Muscle Growth & Protection
Actively Recruiting
Therapeutic Approach: Improving Muscle Growth & Protection
Variant Requirement: Duchenne - No specific requirement
Industry/Sponsor: Satellos Bioscience, Inc.
Ambulation: Ambulatory
Steroid Use: No specific requirement
Eligible Sexes: Male
Clinical Trial NCT ID: NCT07287189
Next Steps:

Registry Study to Observe Long-term Safety of Vamorolone (AGAMREE®) in Patients With Duchenne Muscular Dystrophy-SUMMIT

Actively Recruiting
2+ Years
Observational
Actively Recruiting
Therapeutic Approach: Observational
Variant Requirement: Duchenne - No specific requirement
Industry/Sponsor: Catalyst Pharmaceuticals, Inc.
Ambulation: No specific requirement
Steroid Use: Currently on treatment with Vamorolone (AGAMREE)
Eligible Sexes: Male
Clinical Trial NCT ID: NCT06564974
Next Steps:

The Baby Duchenne Study: Characterizing Developmental and Clinical Outcomes in the First Three Years in Children With Duchenne Muscular Dystrophy

Actively Recruiting
0 Days to <3 Years
Observational
Actively Recruiting
Therapeutic Approach: Observational
Variant Requirement: Duchenne - No specific requirement
Industry/Sponsor: University of Rochester
Ambulation: No specific requirement
Steroid Use: No specific requirement
Eligible Sexes: Male
Clinical Trial NCT ID: NCT07092540
Location: United States
Next Steps:

The Duchenne Registry

Actively Recruiting
All ages
Observational
Actively Recruiting
Therapeutic Approach: Observational
Variant Requirement: No specific requirement
Industry/Sponsor: Parent Project Muscular Dystrophy
Ambulation: Either
Steroid Use: No specific requirement
Eligible Sexes: All
Clinical Trial NCT ID: NCT02069756
Next Steps:

Trial of Cell Based Therapy for DMD

Actively Recruiting
18+ Years
Improving Muscle Growth & Protection
Actively Recruiting
Therapeutic Approach: Improving Muscle Growth & Protection
Variant Requirement: Duchenne - No specific requirement
Industry/Sponsor: University of Minnesota
Ambulation: Non-ambulatory
Steroid Use: No specific requirement
Eligible Sexes: All
Clinical Trial NCT ID: NCT06692426
Location: United States
Next Steps:

Urinary Titin Biomarker in DMD

Actively Recruiting
2 Years to 10 Years
Observational
Actively Recruiting
Therapeutic Approach: Observational
Variant Requirement: No specific requirement
Industry/Sponsor: Children's Hospital of Philadelphia
Ambulation: Ambulatory
Steroid Use: No specific requirement
Eligible Sexes: Male
Clinical Trial NCT ID: NCT07332013
Location: United States
Next Steps:

Vasodilator and Exercise Study for DMD (VASO-REx)

Actively Recruiting
6+ Years
Improving Muscle Growth & Protection
Actively Recruiting
Therapeutic Approach: Improving Muscle Growth & Protection
Variant Requirement: Duchenne - No specific requirement
Industry/Sponsor: University of Florida
Ambulation: Ambulatory
Steroid Use: Stable course of steroids
Eligible Sexes: Male
Clinical Trial NCT ID: NCT06290713
Location: United States

Wearable Technology to Evaluate Hyperglycemia and HRV in DMD - Longitudinal Aim

Actively Recruiting
10+ Years
Observational
Actively Recruiting
Therapeutic Approach: Observational
Variant Requirement: Duchenne - No specific requirement
Industry/Sponsor: Vanderbilt University Medical Center
Ambulation: No specific requirement
Steroid Use: No specific requirement
Eligible Sexes: Male
Clinical Trial NCT ID: NCT06093100
Location: United States
Next Steps:

A Gene Transfer Therapy Study to Evaluate the Safety and Efficacy of Delandistrogene Moxeparvovec (SRP-9001) in Non-Ambulatory and Ambulatory Participants With Duchenne Muscular Dystrophy (DMD)

Active, Not Recruiting
8 Years to 18+ Years
Restoring or Replacing Dystrophin
Active, Not Recruiting
Therapeutic Approach: Restoring or Replacing Dystrophin
Variant Requirement: Duchenne - Excluding any deletion of exon 8 and/or 9
Industry/Sponsor: Sarepta Therapeutics, Inc.
Ambulation: Cohort 1 - Non-ambulatory; Cohort 2 - Ambulatory
Steroid Use: Stable course of steroids
Eligible Sexes: Male
Clinical Trial NCT ID: NCT05881408
Next Steps:

A Study of Deramiocel (CAP-1002) in Ambulatory and Non-Ambulatory Patients With Duchenne Muscular Dystrophy

Active, Not Recruiting
10+ Years
Improving Muscle Growth & Protection
Active, Not Recruiting
Therapeutic Approach: Improving Muscle Growth & Protection
Variant Requirement: Duchenne - No specific requirement
Industry/Sponsor: Capricor Inc.
Ambulation: No specific requirement
Steroid Use: Stable course of steroids
Eligible Sexes: Male
Clinical Trial NCT ID: NCT05126758
Location: United States
Next Steps:

A Study of EDG-5506 in Children With Duchenne Muscular Dystrophy (LYNX)

Active, Not Recruiting
4 Years to <10 Years
Improving Muscle Growth & Protection
Active, Not Recruiting
Therapeutic Approach: Improving Muscle Growth & Protection
Variant Requirement: Duchenne - No specific requirement
Industry/Sponsor: Edgewise Therapeutics, Inc.
Ambulation: Ambulatory
Steroid Use: Cohorts 1,2,3,4,5 - Stable course of steroids; Cohort 2NS - No steroids within 6 months
Eligible Sexes: Male
Clinical Trial NCT ID: NCT05540860
Location: United States
Next Steps:

A Study to Assess the Efficacy and Safety of Satralizumab in Duchenne Muscular Dystrophy

Active, Not Recruiting
8 Years to <18 Years
Enhancing Bone Health
Active, Not Recruiting
Therapeutic Approach: Enhancing Bone Health
Variant Requirement: Duchenne - No specific requirement
Industry/Sponsor: Hoffmann-La Roche
Ambulation: Group 1 - No specific requirement, Group 2 - Ambulatory
Steroid Use: Stable course of steroids
Eligible Sexes: Male
Clinical Trial NCT ID: NCT06450639
Next Steps:

A Study to Compare Safety and Efficacy of High Doses of Eteplirsen in Participants With Duchenne Muscular Dystrophy (MIS51ON)

Active, Not Recruiting
4 Years to <14 Years
Restoring or Replacing Dystrophin
Active, Not Recruiting
Therapeutic Approach: Restoring or Replacing Dystrophin
Variant Requirement: Duchenne - amenable to exon 51 skipping
Industry/Sponsor: Sarepta Therapeutics, Inc.
Ambulation: Ambulatory
Steroid Use: Stable course of steroids
Eligible Sexes: Male
Clinical Trial NCT ID: NCT03992430
Next Steps:

Characterization of Clinical Skeletal and Cardiac Impairment in Carriers of DMD and BMD

Active, Not Recruiting
18+ Years
Observational
Active, Not Recruiting
Therapeutic Approach: Observational
Variant Requirement: Known female carrier and/or mother of a child with Duchenne or Becker
Industry/Sponsor: Nationwide Children's Hospital
Ambulation: No specific requirement
Steroid Use: No current steroid treatment
Eligible Sexes: Female
Clinical Trial NCT ID: NCT02972580
Location: United States
Next Steps:

Defining Endpoints in Becker Muscular Dystrophy

Active, Not Recruiting
6+ Years
Observational
Active, Not Recruiting
Therapeutic Approach: Observational
Variant Requirement: Becker - Variant requirement varies by cohort
Industry/Sponsor: Virginia Commonwealth University
Ambulation: Varies by cohort
Steroid Use: No specific requirement
Eligible Sexes: Male
Clinical Trial NCT ID: NCT05257473
Next Steps:

Long-term Use of Viltolarsen in Boys With Duchenne Muscular Dystrophy in Clinical Practice (VILT-502)

Active, Not Recruiting
4+ Years
Restoring or Replacing Dystrophin
Active, Not Recruiting
Therapeutic Approach: Restoring or Replacing Dystrophin
Variant Requirement: Duchenne - Amenable to exon 53 skipping
Industry/Sponsor: NS Pharma, Inc.
Ambulation: No specific requirement
Steroid Use: Stable course of steroids
Eligible Sexes: Male
Clinical Trial NCT ID: NCT04687020
Next Steps:

Microdystrophin Gene Transfer Study in Adolescents and Children With DMD

Active, Not Recruiting
4 Years to <18 Years
Restoring or Replacing Dystrophin
Active, Not Recruiting
Therapeutic Approach: Restoring or Replacing Dystrophin
Variant Requirement: Duchenne - No specific requirement
Industry/Sponsor: Solid Biosciences Inc.
Ambulation: Ambulatory for children, non-ambulatory for adolescents
Steroid Use: Stable course of steroids
Eligible Sexes: Male
Clinical Trial NCT ID: NCT03368742
Location: United States
Next Steps:

NS-050/NCNP-03 in Boys With DMD (Meteor50)

Active, Not Recruiting
4 years to <16 years
Restoring or Replacing Dystrophin
Active, Not Recruiting
Therapeutic Approach: Restoring or Replacing Dystrophin
Variant Requirement: Duchenne - amenable to exon 50 skipping
Industry/Sponsor: NS Pharma, Inc.
Ambulation: Ambulatory
Steroid Use: Stable course of steroids
Eligible Sexes: Male
Clinical Trial NCT ID: NCT06053814
Next Steps:

Open-label Extension of the HOPE-2 Trial

Active, Not Recruiting
10+ years
Improving Muscle Growth & Protection
Active, Not Recruiting
Therapeutic Approach: Improving Muscle Growth & Protection
Variant Requirement: Duchenne - excludes exon 44 skip-amenable or deletion of exons 3-7
Industry/Sponsor: Capricor Inc.
Ambulation: No specific requirement
Steroid Use: No specific requirement
Eligible Sexes: All
Clinical Trial NCT ID: NCT04428476
Location: United States
Next Steps:

Ph2 Open-label Study of AOC 1044 in Duchenne Muscular Dystrophy Participants With Mutations Amenable to Exon44 Skipping (EXPLORE44OLE)

Active, Not Recruiting
7 Years to <28 Years
Restoring or Replacing Dystrophin
Active, Not Recruiting
Therapeutic Approach: Restoring or Replacing Dystrophin
Variant Requirement: Duchenne - Amenable to exon 44 skipping
Industry/Sponsor: Avidity Biosciences, Inc.
Ambulation: No specific requirement
Steroid Use: No specific requirement
Eligible Sexes: Male
Clinical Trial NCT ID: NCT06244082
Location: United States
Next Steps:

Phase 2 Study of EDG-5506 in Becker Muscular Dystrophy (GRAND CANYON)

Active, Not Recruiting
12 Years to <51 Years
Improving Muscle Growth & Protection
Active, Not Recruiting
Therapeutic Approach: Improving Muscle Growth & Protection
Variant Requirement: Becker - No specific requirement
Industry/Sponsor: Edgewise Therapeutics, Inc.
Ambulation: Ambulatory
Steroid Use: Not currently using
Eligible Sexes: Male
Clinical Trial NCT ID: NCT05291091
Next Steps:

Phase 2 Study of EDG-5506 in Children and Adolescents With Duchenne Muscular Dystrophy Previously Treated With Gene Therapy

Active, Not Recruiting
6 Years to <18 Years
Improving Muscle Growth & Protection
Active, Not Recruiting
Therapeutic Approach: Improving Muscle Growth & Protection
Variant Requirement: Duchenne - No specific requirement
Industry/Sponsor: Edgewise Therapeutics, Inc.
Ambulation: Ambulatory
Steroid Use: Stable course of steroids
Eligible Sexes: Male
Clinical Trial NCT ID: NCT06100887
Location: United States
Next Steps:

Safety, Tolerability, Pharmacodynamic, Efficacy, and Pharmacokinetic Study of DYNE-251 in Participants With Duchenne Muscular Dystrophy Amenable to Exon 51 Skipping

Active, Not Recruiting
4 Years to <17 Years
Restoring or Replacing Dystrophin
Active, Not Recruiting
Therapeutic Approach: Restoring or Replacing Dystrophin
Variant Requirement: Duchenne - amenable to exon 51 skipping
Industry/Sponsor: Dyne Therapeutics
Ambulation: Ambulatory or non-ambulatory <2 years
Steroid Use: Stable course of steroids
Eligible Sexes: Male
Clinical Trial NCT ID: NCT05524883
Next Steps:

Single Escalating Dose Pilot Trial of Canakinumab (ILARIS®) in Duchenne Muscular Dystrophy

Active, Not Recruiting
2+ Years
Reducing Inflammation
Active, Not Recruiting
Therapeutic Approach: Reducing Inflammation
Variant Requirement: Duchenne - No specific requirement
Industry/Sponsor: Children's National Research Institute
Ambulation: Ambulatory
Steroid Use: Steroid-naive
Eligible Sexes: Male
Clinical Trial NCT ID: NCT03936894
Location: United States
Next Steps:

Tadalafil as Adjuvant Therapy for DMD

Active, Not Recruiting
7 Years to <13 Years
Improving Muscle Growth & Protection
Active, Not Recruiting
Therapeutic Approach: Improving Muscle Growth & Protection
Variant Requirement: Duchenne - No specific requirement
Industry/Sponsor: University of Florida
Ambulation: Ambulatory
Steroid Use: No specific requirement
Eligible Sexes: Male
Clinical Trial NCT ID: NCT05195775
Location: United States
Next Steps:

Sodium/Glucose Cotransporter-2 Inhibitors (SGLT2i) Therapy in Duchenne Cardiomyopathy

Not Yet Recruiting
8 to <19 Years
Improving Heart Function
Not Yet Recruiting
Therapeutic Approach: Improving Heart Function
Variant Requirement: Duchenne - No specific requirement
Industry/Sponsor: Riley Children's Hospital
Ambulation: No specific requirement
Steroid Use: No specific requirement
Eligible Sexes: Male
Clinical Trial NCT ID: NCT07172971
Location: United States
Next Steps: