Thalassemia Stocks List

Thalassemia Stocks Recent News

Date Stock Title
May 10 BLUE bluebird bio Inc (BLUE) Q1 2024 Earnings: Surpasses Revenue Expectations with Strategic Focus ...
May 10 BLUE bluebird (BLUE) Tops on Q1 Sales, Zynteglo Progresses Well
May 9 BLUE Bluebird bio spikes even as CRISPR leads in gene therapy rollout
May 9 FULC Fulcrum Therapeutics to Participate in Upcoming May Conferences
May 9 BLUE bluebird bio reports Q1 results
May 9 BLUE bluebird bio Reports First Quarter 2024 Results and Highlights Operational Progress and 2024 Guidance
May 9 BEAM Beam Therapeutics Inc. (NASDAQ:BEAM) Just Reported First-Quarter Earnings: Have Analysts Changed Their Mind On The Stock?
May 8 BLUE bluebird bio Q1 2023 Earnings Preview
May 8 BEAM Beam Therapeutics (BEAM) Q1 Loss Narrows, Revenues Miss
May 8 FULC Fulcrum Therapeutics Announces Publication of Results from Phase 2b Clinical Trial of Losmapimod in Facioscapulohumeral muscular dystrophy (ReDUX4) in The Lancet Neurology
May 8 BEAM Beam Therapeutics to Participate in RBC Capital Markets 2024 Global Healthcare Conference
May 8 BEAM Beam Therapeutics Inc (BEAM) Reports Q1 2024 Earnings: A Detailed Overview
May 7 ALNY Alnylam Issues 2023 Corporate Responsibility Report
May 7 BEAM Beam Therapeutics GAAP EPS of -$1.21 beats by $0.23, revenue of $7.41M misses by $7.25M
May 7 BEAM Beam Therapeutics Reports Pipeline Updates and First Quarter 2024 Financial Results
May 6 BLUE bluebird reports first commercial cell collection for Lyfgenia
May 6 FULC Fulcrum Therapeutics to Host First Quarter 2024 Financial Results Conference Call and Webcast on Monday, May 13, 2024, at 8:00 a.m. ET
May 6 BLUE bluebird bio Announces First Quarter 2024 Results Call Date and Upcoming Investor Events
May 6 BLUE bluebird bio Announces Completion of First Cell Collection for LYFGENIAâ„¢ Gene Therapy
Thalassemia

Thalassemias are inherited blood disorders characterized by decreased hemoglobin production. Symptoms depend on the type and can vary from none to severe. Often there is mild to severe anemia (low red blood cells or hemoglobin). Anemia can result in feeling tired and pale skin. There may also be bone problems, an enlarged spleen, yellowish skin, and dark urine. Slow growth may occur in children.Thalassemias are genetic disorders inherited from a person's parents. There are two main types, alpha thalassemia and beta thalassemia. The severity of alpha and beta thalassemia depends on how many of the four genes for alpha globin or two genes for beta globin are missing. Diagnosis is typically by blood tests including a complete blood count, special hemoglobin tests, and genetic tests. Diagnosis may occur before birth through prenatal testing.Treatment depends on the type and severity. Treatment for those with more severe disease often includes regular blood transfusions, iron chelation, and folic acid. Iron chelation may be done with deferoxamine, deferasirox or deferiprone. Occasionally, a bone marrow transplant may be an option. Complications may include iron overload from the transfusions with resulting heart or liver disease, infections, and osteoporosis. If the spleen becomes overly enlarged, surgical removal may be required. Thalassemia patients who do not respond well to blood transfusions can take hydroxyurea or thalidomide, and sometimes a combination of both. Hydroxyurea is the only FDA approved drug for thalassemia. Patients who took 10 mg/kg of hydroxyurea everyday for a year had significantly higher hemoglobin levels and it was a well-tolerated treatment for patients who did not respond well to blood transfusions. Another hemoglobin-inducer includes thalidomide, although it has not been tested in a clinical setting. The combination of thalidomide and hydroxyurea resulted in hemoglobin levels increasing significantly in transfusion-dependent and non-transfusion dependent patients As of 2015, thalassemia occurs in about 280 million people, with about 439,000 having severe disease. It is most common among people of Italian, Greek, Middle Eastern, South Asian, and African descent. Males and females have similar rates of disease. It resulted in 16,800 deaths in 2015, down from 36,000 deaths in 1990. Those who have minor degrees of thalassemia, similar to those with sickle-cell trait, have some protection against malaria, explaining why they are more common in regions of the world where malaria exists.

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