Found 107 Muscular Dystrophy trials
A listing of Muscular Dystrophy medical research trials actively recruiting patient volunteers. Search for closest city to find more detailed information on a research study in your area.
Featured study
This study investigates hereditary transthyretin-mediated amyloid polyneuropathy, focusing on evaluating the safety and efficacy of an investigational medication for treating this condition.Who can participate: Adults aged 18 to 82 with Stage 1 or Stage 2 Familial Amyloid Polyneuropathy, a genetic mutation in the TTR gene, and symptoms of neuropathy can …
Featured study
The objective of this clinical trial is to assess whether ladarixin treatment is effective in preserving -cell function and delaying the progression of T1D in adolescent and adult patients. The safety of ladarixin in the specific clinical setting will be also evaluated.
Featured study
For subjects age 18 years and older who are at an increased risk for HCC and for whom HCC surveillance is appropriate, including subjects with cirrhosis or non-cirrhotic subjects with Hepatitis B. Approximately 3,000 subjects are expected to be enrolled, comprised of approximately 2,222 ultrasound surveillance subjects and 778 subjects …
Featured study
The purpose of this study is to investigate the collection and storage of biological samples for conditions such as COVID-19, cancer, diabetes, and heart disease.This study evaluates how biological samples and health information can be used in research to enhance understanding and treatment of various conditions.Who can participate: Adults over …
Fibrotic interstitial lungs diseases (ILD) are a group of chronic and rare diseases characterized by an altered blood oxygenation in the lung, leading to dyspnea and physical limitation. Physical training is efficient to fight vicious circle of physical deconditioning observed in ILD. Eccentric cycling is an interesting modality of endurance …
The objectives of the study are to determine the efficacy and safety of Synergo® RITE and mitomycin C (MMC) in the treatment of carcinoma in situ (CIS) non-muscle invasive bladder cancer in adult patients who didn't respond to 2 courses of treatment with Bacillus Calmette-Guérin (BCG) installations. The Synergo® system …
The POSEIDON study is a multi-center, prospective, single arm, crossover pivotal study enrolling up to 60 pivotal cohort patients and up to 30 additional Roll-in patients with refractory or recurrent ascites at up to 15 sites. Pivotal cohort patients will undergo a 3-month pre-implant observation phase after enrollment, followed by …
The research study is being conducted to test the safety and tolerability (i.e., possible side effects) of an investigational drug called AL001 and to find out what effects AL001 has on the body. This is for people who have a confirmed progranulin gene mutation that causes frontotemporal dementia (FTD). Study …
The purpose of this study is to determine the safety and efficacy of mitapivat in patients with sickle cell disease. Patients will be randomly assigned (like flipping a coin) in a 1:1:1 ratio to receive 50mg mitapivat, 100mg mitapivat or matched placebo for twice-daily oral administration. This means that you …
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