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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EMA Grants PRIME Designation to CTX001, a Sickle Cell Disease Treatment
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EMA Grants PRIME Designation to CTX001, a Sickle Cell Disease Treatment

CRISPR Therapeutics, in combination with Vertex Pharmaceuticals, has recently announced that the European Medicines Agency (EMA) has granted the Priority Medicines (PRIME) designation for their sickle cell disease (SCD) treatment,…

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CRISPR Editing Appears Effective in Three Patients with Rare Diseases
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CRISPR Editing Appears Effective in Three Patients with Rare Diseases

According to a story from interestingengineering.com, the capabilities of CRIPSR gene editing technology have been creating a buzz in the medical field for years now, with many seeing the technology…

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The Impact of COVID-19 on Patients with Thalassemia and Sickle Cell Disease: A Webinar Review
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The Impact of COVID-19 on Patients with Thalassemia and Sickle Cell Disease: A Webinar Review

  As COVID-19 continues to spread across the globe, more and more people are wondering how this virus might affect them. This is especially true for patients with rare diseases,…

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Investigative Therapy for Sickle Cell Disease Receives Rare Pediatric Disease Designation
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Investigative Therapy for Sickle Cell Disease Receives Rare Pediatric Disease Designation

Sickle Cell Disease  Sickle cell disease is a rare blood disorder. It is progressive, debilitating, and sadly can be life-threatening. The genetic defect leads patients' red blood cells to be…

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New Approach Uses CRISPR-Cas9 to Boost Fetal Hemoglobin Production to Treat Blood Disorders
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New Approach Uses CRISPR-Cas9 to Boost Fetal Hemoglobin Production to Treat Blood Disorders

  An article published earlier this year by Fierce Biotech outlines an approach that was developed by researchers at the Fred Hutchinson Cancer Research Center. Using CRISPR-Cas9’s gene-editing method the researchers were…

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This Failed Clinical Trial Helped Researchers Learn More About Sickle Cell Disease
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This Failed Clinical Trial Helped Researchers Learn More About Sickle Cell Disease

  No healthcare professional wants to hear that a drug being studied in a clinical trial did not meet the endpoints. It means that after countless hours of hard work,…

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A Woman with Sickle Cell Disease Hopes to Become a Doctor to Help Others with the Same Disease

Copyscape score: 9% Deondra Clark was unable to have many of the experiences that other children had. Homecoming, prom, sports, and other childhood activities were not a possibility for her,…

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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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