Clinical Research Directory

A Phase 3 Study to Evaluate the Safety and Efficacy of AOC 1044 (Also Referred to as Delpacibart Zotadirsen) in Participants With DMD With Gene Mutations Amenable to Exon 44 Skipping

A Randomized, Double-blind, Placebo-controlled, Phase 3 Study to Evaluate the Efficacy and Safety of Intravenous AOC 1044 for the treatment of Duchenne Muscular Dystrophy (DMD) with Gene Mutations Amenable to Exon 44 Skipping

A Clinical Study to Assess Safety, Efficacy and Pharmacodynamic Profile of GNT0004 in Duchenne Muscular Dystrophy

GNT0004 is a recombinant adeno-associated viral (AAV) vector gene therapy composed of an AAV8 serotype capsid containing a sequence-optimized gene for a human microdystrophin. Duchenne muscular dystrophy (DMD) is a neuromuscular disorder caused by dystrophin gene mutations leading to absence of functional dystrophin, a key protein that prevents muscle cell damage during physiological contractions. To address the underlying cause of the disease, GNT0004 delivers an optimized dystrophin gene that is capable to produce a shortened but functional dystrophin protein in target tissues (heart and skeletal muscles) in DMD subjects. It is expected to significantly delay or slow disease progression. GNT0004 will be given as a one-time intravenous (IV) infusion to help it reach muscles throughout the body.

Whole-body Vibration Versus Gravity Force Stimulation on Postural Stability in Children With Duchene Muscular Dystrophy

this study aimed to mention Does whole-body vibration training or gravity-force stimulation produce greater improvements in postural stability among children with Duchenne Muscular Dystrophy?

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

This is a prospective observational study conducted to evaluate safety, tolerability, and functional outcomes of patients with DMD newly initiating oral givinostat or having started therapy within 6 months as part of routine clinical care in the US. The study has a planned maximum duration of 5 years for the first enrolled patients, including a 24-month enrollment period and a minimum of 2 years of follow-up.

Multiparametric Ultrafast Ultrasound Biomarkers for Duchenne and Becker Muscular Dystrophies

The purpose of this research study is to determine the potential of a multiparametric ultrasound approach to non-invasively monitor disease progression and to serve as an objective outcome measure for future clinical trials in Duchenne and Becker Muscular Dystrophies. The investigators will compare the muscles of ambulatory or non-ambulatory boys/men with Duchenne and Becker Dystrophies with muscles of healthy age-matched individuals of the same age and monitor disease progression in those with muscular dystrophies over a 12-month year period. The ultrafast ultrasound technology used in this study allows the simultaneous assessment of muscle structure, mechanics and physiology, including stiffness, anisotropy, viscosity, intramuscular fat, muscle volume, and microvascular perfusion. The amount of muscle alteration measured will be related to performance in daily activities, such as walking and muscle strength, in order to identify sensitive and objective markers of disease progression.

Home-Based Tele-Rehabilitation for Respiratory Function in Children With Duchenne Muscular Dystrophy

This study evaluates the feasibility and preliminary clinical effects of a home-based tele-rehabilitation program aimed at improving respiratory function in children with Duchenne Muscular Dystrophy (DMD).

How Can Different Forms of Strengthening Exercises Affect the Muscular Tissue and Its Chemical Markers in Children With Duchenne Muscular Dystrophy

The treatment of patients diagnosed with DMD needs to be multidisciplinary, judicious, and always focused on the patient's well-being. Physiotherapy and occupational therapy are very often standard care. Physiotherapy is regarded as crucial for the success of treatment, since it has achieved good results in the short term, such as maintaining the autonomy of these individuals . Physical activity is another method that has been used to treat DMD. Studies have used physical exercise in individuals with DMD in order to decrease muscle deterioration, muscle contractures, and bone fractures, and to increase the time of functional independence