Rare Disease Patient Registry & Natural History Study - Coordination of Rare Diseases at Sanford...
Rare DisordersUndiagnosed Disorders316 moreCoRDS, or the Coordination of Rare Diseases at Sanford, is based at Sanford Research in Sioux Falls, South Dakota. It provides researchers with a centralized, international patient registry for all rare diseases. This program allows patients and researchers to connect as easily as possible to help advance treatments and cures for rare diseases. The CoRDS team works with patient advocacy groups, individuals and researchers to help in the advancement of research in over 7,000 rare diseases. The registry is free for patients to enroll and researchers to access. Visit sanfordresearch.org/CoRDS to enroll.
The EXPLORE MG Registry for Myasthenia Gravis
Myasthenia GravisThe purpose of this study is to create a Yale University Department of Neurology Myasthenia Gravis (MG) registry that will be used for current and future research projects involving the study of Myasthenia Gravis.
Exploring Outcomes and Characteristics of Myasthenia Gravis 2
Myasthenia GravisThe goal of this prospective observational study is to create a network repository of clinical data and biological samples to help researchers learn more about myasthenia gravis.
The ME&MG Digital Solution for Autonomous Assessment of Myasthenia Gravis
Myasthenia GravisME&MG is a standalone software (digital solution) running on patients smartphones, connected to a web portal for physicians. It is intended to be used as an unsupervised digital self-assessment tool for the monitoring of disabilities in patients living with MG. ME&MG contains digital active tests for the assessment of ptosis, breathing, dysarthria, upper- and lower-limb (arms and legs) weakness, treatment follow-up, and validated e-questionnaires related to daily activities, pain, fatigue, sleep, and depression disorders. The objectives of this study are to validate the accuracy, reliability and reproducibility of the unsupervised at-home self-assessment of symptoms on the patient's smartphone with ME&MG versus the standard in-clinic testing, as well as to evaluate the safety of the solution, its usability and satisfaction.
Safety and Efficacy Study of Eculizumab in Patients With Refractory Generalized Myasthenia Gravis...
Myasthenia GravisThe purpose of this study is to determine whether eculizumab is safe and effective in the treatment of patients with generalized myasthenia gravis despite treatment with various immunosuppressants, such as prednisone, methotrexate, Cellcept, cyclosporine, and cyclophosphamide, that are currently available.
Subcutaneous Ig Maintenance Therapy for Myasthenia Gravis
Myasthenia GravisThe study is being done with patients with Myasthenia Gravis (MG), age 18-80 years, positive acetylcholine receptor antibody, receiving greater than 30mg of prednisone daily. Patients may or may not be receiving anticholinesterase agents. A common treatment for patients with this disease includes the administration of intravenous immunoglobulin (IVIG), which is a plasma protein that is given to help maintain adequate antibody levels to prevent infections and decrease the symptoms of the disease of Myasthenia Gravis. This study is being done to test if giving this medication in a subcutaneous form (into the fat of the abdomen, legs and thighs) will be better tolerated for patients with Myasthenia Gravis.
Efficacy of Prednisone In the Treatment of Ocular Myasthenia
Ocular Myasthenia GravisThe purpose of this study is to evaluate the efficacy and tolerability of prednisone in patients diagnosed with ocular myasthenia. Funding Source - FDA OOPD
Randomized Study of Intravenous Immunoglobulin in Patients With Mild or Moderate Myasthenia Gravis...
Myasthenia GravisOBJECTIVES: I. Determine whether intravenous immunoglobulin is an effective therapy for patients with mild or moderate myasthenia gravis.
Pharmacodynamic Study of CK-2017357 in Patients With Generalized Myasthenia Gravis
Myasthenia GravisThe primary objective of this early-stage clinical study is to demonstrate an effect of single doses of CK-2017357 on measures of skeletal muscle function and fatigability in patients with generalized myasthenia gravis (MG).
An Extension Study of MOM-M281-004 to Evaluate the Safety, Tolerability, and Efficacy of M281 Administered...
Generalized Myasthenia GravisThe purpose of this study is to evaluate the long-term safety and tolerability of M281 in participants with generalized myasthenia gravis (gMG)