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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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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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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The Cure Rare Disease Foundation is Trying to Change the “One Size Fits All” Approach Toward Treatment

The Cure Rare Disease Foundation The Cure Rare Disease Foundation's mission is to increase collaborative efforts between researchers in order to accelerate the development of customized therapies for individuals living with…

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Loma Linda University Cures Their First Sickle Cell Disease Patient with Stem Cell Transplant

LLUCH Loma Linda University Children's Hospital (LLUCH) is one of six hospitals in the University's health system. They are a faith-based system located in Southern California. They are the only…

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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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