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This study is an observational (ie, noninterventional), longitudinal, multicenter, global registry for patients with pyruvate kinase (PK) deficiency, a rare nonspherocytic hemolytic anemia. This Registry will be open for enrollment for 7 years and all enrolled participants will be followed prospectively for a minimum of 2 years, and up to …
The purpose of this study is to evaluate two study medicines (encorafenib plus cetuximab) taken alone or together with standard chemotherapy for the potential treatment of colorectal cancer that: has spread to other parts of the body (metastatic); has a certain type of abnormal gene called "BRAF"; and has not …
This phase II ComboMATCH treatment trial compares the effect of neratinib to the combination of neratinib and palbociclib in treating patients with HER2 positive solid tumors. Neratinib and palbociclib are in a class of medications called kinase inhibitors. They work by blocking the action of an abnormal protein that signals …
This project will test the efficacy of a multi-component behavioral intervention to improve metabolic control among older adolescents and emerging adults (16-21) with T1D, a group with chronic poor metabolic control. This intervention is grounded in self-determination theory which states that a youth who believes their diabetes management is self-directed, …
Background Smith-Lemli-Opitz Syndrome (SLOS) is a genetic disorder. It can cause birth defects and developmental delays. There is no cure for SLOS or other inherited diseases related to cholesterol production or storage. The data gained in this study may help researchers find ways to measure how well future treatments work. …
As part of a post approval commitment, the MFDS has requested a study to characterize safety in patients who are treated with FASENRA for severe asthma with an eosinophilic phenotype by physicians in normal clinical practice settings. This study is designed to confirm or assess the known safety profile or …
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