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Filter by:Lambert-Eaton Myasthenic Syndrome (LEMS) is a rare autoimmune disorder which affects the nerve-muscle junction. The major symptoms of LEMS are progressive muscle weakness. Many patients experience other symptoms like dry mouth or impotence. Congenital Myasthenia (CM) is an inherited disorder with similar affects and symptoms. 3,4-Diaminopyridine (DAP) is an experimental drug that has improved strength in some subjects with (LEMS). There are no other accepted treatments for LEMS and DAP has relatively few side effects.
Compassionate use of Stiripentol in Dravet Syndrome. This is a treatment protocol, not a research study, therefore children will only be monitored on a clinical basis for seizure improvement predominantly by parent and caregiver report.
This protocol has provided 3,4 diaminopyridine (DAP) under a treatment-use IND to patients with congenital myasthenic syndrome (CMS). It is currently closed to enrollment.
The Requesting Physician/Investigator contacts Lilly when, based on their medical opinion, a patient meets the criteria for inclusion in the expanded access program.
The purpose of this study is the examination of Mapisal® versus urea hand-foot cream as prophylaxis for capecitabine-induced hand-foot syndrome (HFS) in patients with gastrointestinal tumors or breast cancer, to assess the efficacy of Mapisal®. Mapisal® is a medical device that is approved for the prophylaxis and treatment of HFS. Initial clinical data and case studies on the treatment and prophylaxis of Caelyx-induced HFS have been impressive. Because the pathomechanism of HFS caused by capecitabine is the same as for Caelyx-induced HFS, it is expected reason that administering Mapisal® should result in a significant reduction of HFS caused by capecitabine. The urea hand-foot cream was selected for the standard arm, because it is used commonly, is accepted by patients, and seems to have a positive influence on the severity of the HFS in the experience of many oncologists.
The patient has failed all other available agents and has intractable epilepsy due to Dravet Syndrome. Stiripentol is highly efficacious in Dravet Syndrome. The overall goals of therapy with Stiripentol are primarily to significantly reduce the frequency and severity of seizures.
Lambert Eaton Myasthenic Syndrome (LEMS) is rare neurological disorder that results in muscle weakness and limited reflex activity. More than half of LEMS cases are associated with a malignancy, usually small cell lung cancer, and tend to progress more quickly than cases not coupled with malignant cells. 3,4diaminopyridine (3,4DAP)is a drug that has been demonstrated to be effective in treating the weakness associated with LEMS as it increases strength and improves autonomic symptoms in LEMS patients. It is not currently approved by the FDA for use in the United States. The investigators plan to use 3,4DAP to treat patients with LEMS here at the Columbia University MDA/ALS Research Center.
Compassionate use of orphan drug 3,4-Diaminopyridine(DAP) in Treatment of Lambert Eaton Myasthenic Syndrome (LEMS). 3,4-DAP is used to decrease the muscle weakness associated with LEMS and hopefully will decrease the need for prednisone and all other therapies that were previously required to control symptoms. How long a patient will take 3,4 DAP depends upon if he/she is seeing benefits from the medication or experiencing side effects that will prevent them from continuation in the study.
The purpose of this study is to provide Decitabine to patients with Myelodysplastic syndrome (MDS) of all FAB (French-American-British) subtypes and Intermediate-1, Intermediate-2, and High-Risk International Prognostic Scoring System groups, including both previously treated and untreated patients.
The purpose of the study is to provide expanded access (compassionate use) of 3,4 diaminopyridine to patients with Lambert-Eaton myasthenic syndrome (LEMS).