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.
This research is being done to study a new way to treat FTD in patients who have the GRN mutation. This process is called “gene therapy”. Gene therapy is an experimental technique that aims to replace the gene that is not working properly with a version of the gene that …
TEGSEDI (also known as inotersen) is a medicine that has been approved in the United States, Europe and Canada to treat polyneuropathy (nerve damage) caused by hereditary transthyretin amyloidosis (hATTR-PN) in adults. hATTR-PN is a rare genetic disease that worsens over time if left untreated. This research study will examine …
This research study is being done at the request of the US Food & Drug Administration (FDA) to collect additional safety information for the commercially available drug TEGSEDI. This research study is being conducted to examine any changes in health or side effects occurring within 24 hours following treatment with …
The Exablate Model 4000 Type-1.0/1.1 Neuro system involves using an advanced, non-invasive technique currently approved by the Food and Drug Administration (FDA) to treat one side of your brain in a procedure called a thalamotomy (lesion the thalamus). This procedure involves performing an ablation (lesion) of an area of the …
The research study is being conducted to investigate if the study drug BIIB059 can reduce disease activity and inflammation in people with active Cutaneous Lupus Erythematosus compared to a placebo. Participants will complete the following research procedures: demography and medical history, previous CLE therapy, SARS-CoV-2 (COVID-19) test, chest X-ray, skin …
The purpose of this protocol is to monitor the rate of recurrence in patients who undergo cancer surgery. Patient diagnosed with solid tumor cancers have a high rate of recurrence when surgery is a treatment. The high rates of recurrence suggest that surgeons are unable to completely detect and remove …
To identify clinically evident rejection events with consistency and accuracy using Computer Assisted Cardiac Histologic Evaluation (CACHE) system that surpasses that of the standard ISHLT rejection grading by trained pathologists
The research team aims to examine whether amantadine can help patients recover from stroke. This will be a blinded randomized clinical trial. We will randomize patients post-ischemic or hemorrhagic stroke either to the placebo arm or amantadine. Patients will be on study drug or placebo for 1 month but will …
The target population are patients at Penn Medicine undergoing elective bariatric surgery; and are additionally participating in POW-R study ( Protocol #843280) Patients will be recruited to undergo liver wedge and visceral adipose tissue biopsy and provide blood samples. Liver samples will be processed for RNA, metabolomics, and biochemical and …
The primary objective of this study is to characterize the safety, tolerability, dose limiting toxicities (DLTs), and maximum tolerated dose (MTD) or maximum administered dose (MAD) (if no MTD is defined) of MGD013 when administered intravenously (IV) every 2 weeks to patients with unresectable, locally advanced or metastatic cancers.
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