Clinical Trial Results Look Good for Possible Treatment for Transfusion Dependent Beta Thalassemia
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Clinical Trial Results Look Good for Possible Treatment for Transfusion Dependent Beta Thalassemia

According to a story from Market Screener, the biopharmaceutical company Orchard Therapeutics recently announced that the results of a recent clinical trial testing the company' investigational product OTL-300 were encouraging.…

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The Latest in St. Jude Research on Blood Cancers and Blood Disorders
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The Latest in St. Jude Research on Blood Cancers and Blood Disorders

According to a story from EurekAlert!, the 60th Annual Meeting of the American Society of Hematology featured a number of different presentations of the latest research from St. Jude Children's…

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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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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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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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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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