Publications by authors named "Sara Tama-Shekan"
Discov Med
September 2024
Article Synopsis
- Systemic light-chain (AL) amyloidosis is a rare condition caused by misfolded immunoglobulin light chains that accumulate in organs, leading to dysfunction and failure, especially when the heart is involved.*
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- Treatment primarily focuses on eliminating the abnormal plasma cell growth, with the current first-line therapy being a combination of four drugs known as DaraCyborD; some patients may require stem cell transplants if this approach is insufficient.*
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- Recent advancements include the use of monoclonal antibodies, like CAEL 101 and Birtamimab, aimed at breaking down amyloid deposits in affected organs, currently being tested in phase 3 trials for patients with significant cardiac involvement.*
Article Synopsis
- Pyruvate kinase (PK) deficiency is an inherited condition causing chronic hemolytic anemia, with symptoms like fatigue, jaundice, and gallstones, typically managed with transfusions and other supportive therapies.
- Mitapivat is a newly approved oral medication that activates RBC PK enzymes and has shown positive results in some patients by improving hemoglobin levels and reducing transfusion needs, though not all respond to it.
- The response to mitapivat varies based on individual genetic factors, suggesting that both clinical symptoms and genetic background should be considered before starting treatment.
