Downsyndrome (DS) occurs in roughly 1 out of 700 births in the U.S. Half of all children with Downsyndrome have congenital heart disease (CHD), often requiring corrective heart surgery. Even though CHD in children without DS is known…
This study is for patients with Lambert-Eaton Myasthenic Syndrome (LEMS). The purpose of this study is to provide doctors and their patients access to amifampridine phosphate therapy for their patients with LEMS, CMS or downbeat nystagmus until the product becomes commercially available. The…
The purpose of this study is to test the safety and effectiveness of study drug PLX51107 on patients with advanced malignancies such as solid tumors, lymphoma, AML or MDS. The participants in this study will be the first people to receive PLX51107. PLX51107 works by targeting and inhibiting certain activities within cells that promote tumor growth. By…
The purpose of the Main study is to test the overall effects of E7777 on you and your illness. It is not known what effect E7777 will have on your illness or what the side effects will be. This study will look at the effect E7777 has on your body and your disease. It will also look at how your body absorbs, distributes, breaks down…