Presentations Demonstrate Promising Advances in Blood Cancers and Blood Disorders

According to a story from PR Newswire, presentations at the 60th annual American Society of Hematology Meeting and Exposition are highlighting the results of four large scale clinical trials that…

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Working to Improve Gene Therapy for Adrenoleukodystrophy and Other Rare Conditions

Rare disease research is often underrepresented in the field of science. However, recent developments have made researchers more interested in studying this group of diseases. For instance, investments in gene…

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Fast Track Awarded to a Drug for Anaemia in Patients with Beta-Thalassemia
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Fast Track Awarded to a Drug for Anaemia in Patients with Beta-Thalassemia

The United States Food and Drug Administration has awarded Fast Track designation to an investigational drug called PTG-300 for treating chronic anaemia caused by ineffective erythropoiesis in patients who have…

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A Marketing Application for the Gene Therapy LentiGlobin as a Treatment for Beta-Thalassemia is Being Reviewed in Europe

According to a recent press release from Bluebird Bio, the European Medicines Agency has accepted and will review their marketing authorization application (MAA) for the investigational gene therapy LentiGlobin™ as…

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Experimental Treatment for Anemia Associated With Beta Thalassemia Gets Fast Track Designation
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Experimental Treatment for Anemia Associated With Beta Thalassemia Gets Fast Track Designation

According to a story from PR Newswire, the drug developer Protagonist Therapeutics recently announced that its investigational therapy PTG-300 has earned Fast Track designation from the US Food and Drug…

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Is There a Genetic Condition in Your Family?
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Is There a Genetic Condition in Your Family?

Is there a genetic condition in your family? Are you concerned about the potential for passing this on to your children one day? Do you know if you are a…

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The FDA has placed an Investigational Drug For Sickle Cell Disease on Clinical Hold
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The FDA has placed an Investigational Drug For Sickle Cell Disease on Clinical Hold

The Investigational New Drug Application for the experimental drug CTX001 for treating sickle cell disease has been put on clinical hold, announced Vertex Pharmaceuticals Incorporated and CRISPR Therapeutics. This hold…

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Gene Therapy Could Put an End to Blood Transfusions for Beta Thalessemia Patients
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Gene Therapy Could Put an End to Blood Transfusions for Beta Thalessemia Patients

According to a story from empr.com, a recent study revealed that gene therapy could allow patients with beta thalessemia to stop having to get regular blood transfusions. The gene therapy…

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Thalassemia and Gaucher May Be Linked Suggests A Surprising New Case Study
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Thalassemia and Gaucher May Be Linked Suggests A Surprising New Case Study

A new case study suggests that the blood disorder, thalassemia, may be present in people with Gaucher disease. Both diagnosis and treatment become more complicated if this hypothesis proves true.…

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Study of LJPC-401 Treatment for Hereditary Hemochromatosis Launches into Phase II
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Study of LJPC-401 Treatment for Hereditary Hemochromatosis Launches into Phase II

Hereditary hemochromatosis is a genetic form of a condition in which the body builds up too much iron. To read more about hemochromatosis, click here. La Jolla Pharmaceutical Company has…

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Is it Ethical? Should Human Embryos Be Used to Prevent Genetic Diseases Like Beta Thalassemia?
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Is it Ethical? Should Human Embryos Be Used to Prevent Genetic Diseases Like Beta Thalassemia?

How far would you go to eradicate a disease that impacts millions of lives? Would you cross the ethical line if it meant ending a source of suffering forever? Or…

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What to Eat in the World of Atkins, Gluten-Free, and Paleo Diets
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What to Eat in the World of Atkins, Gluten-Free, and Paleo Diets

Don’t eat that! Eat this instead. Some diets recommend low fat. Some endorse high protein. Some forbid carbohydrates. Some praise those same carbohydrates. Some limit fiber. It’s so confusing figuring…

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Major Gene Therapy Progress for β-thalassemia Major
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Major Gene Therapy Progress for β-thalassemia Major

β-thalassemia major is a rare blood condition that necessitates a lifetime of blood transfusions. Despite advancements in treatments, the perfect cure has yet to be found. Bluebird Bio, Inc. is…

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This Drug May Reduce Your Dependence on Transfusions
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This Drug May Reduce Your Dependence on Transfusions

Celgene Corporation, partnered with Acceleron Pharma Inc., announced at the beginning of this month that target enrollment of myelodysplastic syndromes (MDS) and beta thalassemia patients for the MEDALIST and BELIEVE…

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Hemochromatosis – When the Body Can’t Get the Iron Out
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Hemochromatosis – When the Body Can’t Get the Iron Out

Most of us know that not having enough iron in the blood results in disorders like anemia, fatigue, low energy… I first became aware of “iron-poor blood,” or anemia, when…

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