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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New Treatment Shows Benefits for Patients with Sickle Cell Disease and Beta Thalassemia

  New knowledge of the genes that affect sickle cell disease and beta thalassemia has allowed for advances in treatments for these diseases. Advances in technology have also helped with…

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Immunizations and Rare Diseases: A Resource

The Immunization Action Coalition (IAC) Express Newsletter reports that 981 cases of measles have been reported so far this year, with 41 new cases added just last week.  Rare disease kids…

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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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Extremely Positive Results Announced from Interim Analysis of Phase 2 Clinical Trial for Hemochromatosis

Hereditary Hemochromatosis Hereditary hemochromatosis (HH) is a rare disease caused by hepcidin deficiency or hepcidin insensitivity. Hepcidin naturally regulates iron absorption/distribution in the body. Without hepcidin, HH patients suffer from…

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