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Genetic Studies of Patients and Their Families With Inherited Cardiovascular and Neuromuscular Diseases.
We are studying the genetics of human cardiovascular and neuromuscular disease. There are many different genetic regions that have been associated with the dev…
Open Label, Long-term Safety, Tolerability, and Efficacy Study of GIVINOSTAT in All DMD Patients Who Have Been Previously Treated in One of the GIVINOSTAT Studies
This is an open label, long-term safety, tolerability, and efficacy study of GIVINOSTAT in all DMD (Duchenne's muscular dystrophy) patients who have been previ…
A Phase I/II Trial Evaluating Long-Term Use of a Pediatric Robotic Exoskeleton (P.REX/Agilik) to Improve Gait in Children With Movement Disorders
Background: People with cerebral palsy, spina bifida, muscular dystrophy, or spinal cord injury often have muscle weakness and problems controlling how their …
Magnetic Resonance Imaging and Biomarkers for Muscular Dystrophy
The purpose of this research study is to determine the potential of magnetic resonance imaging, spectroscopy, and whole body imaging to monitor disease progres…
Wearable Technology to Evaluate Hyperglycemia and Heart Rate Variability in Duchenne Muscular Dystrophy
Duchenne Muscular Dystrophy (DMD) is an X-linked disorder that causes muscle wasting, cardiopulmonary failure, and premature death. Heart failure is a leading …
National Registry of Myotonic Dystrophy and Facioscapulohumeral Muscular Dystrophy Patients and Family Members
Myotonic dystrophy (DM) and facioscapulohumeral muscular dystrophy (FSHD) are inherited disorders characterized by progressive muscle weakness and loss of musc…
A Phase 1b/2a, Open-label, Dose Escalation Study to Evaluate the Safety and Clinical Activity of Intramuscular Doses of BB-301 Administered to Subjects With Oculopharyngeal Muscular Dystrophy With Dysphagia
Subjects who have enrolled in the oculopharyngeal muscular dystrophy (OPMD) natural history study (Study BNTC-OPMD-NH-001) and have completed at least 6 months…
An Open-Label, Systemic Gene Delivery Study Using Commercial Process Material to Evaluate the Safety of and Expression From SRP-9001 in Subjects With Duchenne Muscular Dystrophy (ENDEAVOR)
This is an open-label gene transfer therapy study evaluating the safety of and expression from delandistrogene moxeparvovec in participants with DMD. The maxim…
A Phase 2 Study to Assess the Efficacy, Safety, Tolerability, and Pharmacokinetics of NS-089/NCNP-02 in Boys With Duchenne Muscular Dystrophy (DMD)
This is a Phase 2, open-label, multi-center, 2-part study of NS-089/NCNP-02 administered by weekly IV infusion to ambulant boys aged ≥4 to \<15 years with DMD …
A Phase 2a, Randomized, Double-Blind, Placebo-Controlled Dose Comparison and Exploratory Efficacy Study of Orally Administered SAT-3247 in Ambulatory DMD Patients
Phase 2a trial of SAT-3247 in ambulatory DMD patients aged ≥ 7 and \< 10 years. The trial will study two doses of SAT-3247 in a randomized, double-blind, place…
Establishing Clinical Trial Readiness for Children 0-5 Years With Congenital Muscular Dystrophy Secondary to LAMA2 Mutations (READY CMD LAMA2)
The goal of this observational study is to understand how young children with LAMA2-related dystrophy move and change over time. We will also learn about how t…
Effects of Whole-body Electrical Muscle Stimulation Exercise on Adults withNeuromuscular Disease
This single-arm pilot study evaluates the effects of whole-body electrical muscle stimulation (WB-EMS) exercise on neuromuscular and physical function in adult…