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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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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CRISPR/Cas9 Looks Promising for Transfusion Dependent Sickle Cell Disease and Beta Thalassemia Patients 
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CRISPR/Cas9 Looks Promising for Transfusion Dependent Sickle Cell Disease and Beta Thalassemia Patients 

   CRISPR/Cas9 technology reached another milestone with encouraging results in two recent trials of CTX001. An article in Pharmaceutical Technology carried an announcement by CRISPR Therapeutics and Vertex Pharmaceuticals about…

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An Oklahoma Medical Research Foundation Scientist Played a Critical Role in Developing a New Sickle Cell Disease Treatment
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An Oklahoma Medical Research Foundation Scientist Played a Critical Role in Developing a New Sickle Cell Disease Treatment

According to a story from The Oklahoman, Dr. Roger McEver, who is a scientist and the Vice President of Research at the Oklahoma Medical Research Foundation (OMRF), played a pioneering…

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New Prime Editing Has The Potential to Search and Replace up to 89% of Genetic Defects
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New Prime Editing Has The Potential to Search and Replace up to 89% of Genetic Defects

  CNN recently covered a story in the publication Nature about a paper explaining base editing (or prime editing). The researchers who created the technology set forth the process of using base…

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CRISPR Gene Editing Promises to Cure 10,000 diseases. Now in Its First Human Clinical Trial. Will it Eventually Deliver?

  Clustered Regularly Interspaced Short Palindromic Repeats is more commonly known as CRISPR. As reported recently in Science News, the CRISPR/Cas9 “molecular scissors” is slated to make its highly anticipated debut…

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Antibodies: The Key to Fighting Sickle Cell Anemia? Approval Could be Just Months Away
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Antibodies: The Key to Fighting Sickle Cell Anemia? Approval Could be Just Months Away

According to a story from PMLive, the US Food and Drug Administration (FDA) has recently begun the priority review process for a new potential therapy from pharmaceutical behemoth Novartis. This…

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Experimental Drug for Sickle Cell Disease Earns Fast Track Designation

According to a story from BioSpace, the biopharmaceutical company Imara, Inc. has recently announced that its experimental product candidate IMR-687 has earned Fast Track designation from the US Food and…

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A Beta Thalassemia Patient will be the First Dosed with CRISPR-Based Gene Therapy in Company Sponsored Trial
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A Beta Thalassemia Patient will be the First Dosed with CRISPR-Based Gene Therapy in Company Sponsored Trial

According to a story from MedCity News, a patient with the rare blood disorder beta thalassemia will be the first patient dosed in a Phase I/II clinical trial involving a…

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