Crizanlizumab for SCD is Coming to England
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Crizanlizumab for SCD is Coming to England

According to The Guardian, England’s National Health Service (NHS) has announced that a new therapeutic option will be available for patients with sickle cell disease (SCD). This treatment, called crizanlizumab,…

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Could Base Editing Treat SCD?
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Could Base Editing Treat SCD?

Have you ever heard of base editing? This relatively new technology within the field of genome editing offers the potential opportunity to treat a variety of genetic conditions. According to…

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Patients Who Received CRISPR Gene Editing for Beta Thalassemia and Sickle Cell Disease Remain Pain Free
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Patients Who Received CRISPR Gene Editing for Beta Thalassemia and Sickle Cell Disease Remain Pain Free

  The initial CRISPR treatment for sickle cell disease was administered to Victoria Gray in July 2019. Victoria, a resident of Forest, Mississippi, was born with sickle cell and has…

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Emmaus Life Sciences Makes Drug For Sickle Cell Disease Free For Those That Can’t Pay
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Emmaus Life Sciences Makes Drug For Sickle Cell Disease Free For Those That Can’t Pay

As reported in PR NewsWire;  in an effort to help the entirety of the sickle cell disease community, Emmaus Life Sciences, Inc has announced a program that will provide their…

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A CRISPR Tool With the Potential to Halt the Progression of Sickle Cell and Beta Thalassemia

According to an NBC AP news item, early results show that CRISPR, a “tool” that alters DNA permanently in blood cells, has the potential to not only stop the progression…

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Use of Tissue Type Plasminogen Activator in Sickle Cell Disease Patients Experiencing Stroke
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Use of Tissue Type Plasminogen Activator in Sickle Cell Disease Patients Experiencing Stroke

by Lauren Taylor from In The Cloud Copy Sickle cell disease or SCD is a type of red blood cell disorder in which the normally round red blood cells are…

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Experimental Treatment for Sickle Cell Disease Earns Orphan Drug Designation in the EU
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Experimental Treatment for Sickle Cell Disease Earns Orphan Drug Designation in the EU

According to a story from Street Insider, the biopharmaceutical company Forma Therapeutics Holdings, Inc., recently announced that its investigational therapy candidate FT-4202 has earned Orphan Drug designation from the European…

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Now Available: FTX-6058 Proof-of-Concept Data for SCD, Beta Thalassemia
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Now Available: FTX-6058 Proof-of-Concept Data for SCD, Beta Thalassemia

For years, clinical-stage biopharmaceutical company Fulcrum Therapeutics ("Fulcrum") has worked to develop therapies for patients with rare genetic disorders and diseases. Somewhat recently, their focus was on treating beta thalassemia…

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