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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Chinese Researcher’s Gene Editing Project Lands Him in Hot Water, But Could it Lead to Answers for Rare Disease Patients in Future?
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Chinese Researcher’s Gene Editing Project Lands Him in Hot Water, But Could it Lead to Answers for Rare Disease Patients in Future?

One Chinese scientist has completed a research project that has shocked many in the medical community. Two twin baby girls born in November of 2018 where the recipients of his…

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The Patriots Raise Awareness for Sickle Cell Anemia and Duchenne Muscular Dystrophy
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The Patriots Raise Awareness for Sickle Cell Anemia and Duchenne Muscular Dystrophy

This past Sunday, December 2nd, during the Patriot's game against the Minnesota Vikings, 20 players wore customized cleats to spread awareness for various philanthropic causes. This is the third year…

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Bioverativ Presents Updates on Treatments and Research for Sickle Cell Anemia and Hemophilia
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Bioverativ Presents Updates on Treatments and Research for Sickle Cell Anemia and Hemophilia

According to a story from Business Wire, the drug developer Bioverativ, Inc., recently announced its intention to present data related to its latest developments in blood disorder therapies. This data…

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A Woman with Sickle Cell Disease Has Created a Foundation to Support Others with the Condition
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A Woman with Sickle Cell Disease Has Created a Foundation to Support Others with the Condition

The Noah’s Ark Foundation For Sickle Cell, an NGO based in Lagos, Nigeria, is working to support people with sickle cell disease. In a recent interview with the Nigerian Tribune,…

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Innovative Program in Greenville, SC Helps Patients With Sickle Cell Anemia
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Innovative Program in Greenville, SC Helps Patients With Sickle Cell Anemia

According to a story from Greenville Online, the Greenville Health System has begun implementing a comprehensive treatment program for patients with sickle cell disease and its most severe and common…

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Genetic Screening Can Prevent Parents from Passing on Genes Linked to Some Diseases
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Genetic Screening Can Prevent Parents from Passing on Genes Linked to Some Diseases

The Young family from Oregon, U.S. have used genetic screening to test their embryos for a variation of the BRCA gene that significantly increases the risk of breast cancer, reports…

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A New Sickle Cell Diseases Drug Has Been Awarded Orphan Drug Status by the US and EU
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A New Sickle Cell Diseases Drug Has Been Awarded Orphan Drug Status by the US and EU

A new drug called Altemia is being developed to treat sickle cell diseases in children, reports BioPortfolio. It has recently been awarded Orphan Drug designation in both the United States…

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The FDA is Beginning to Accept New Endpoints and “Real World” Data in Clinical Trials
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The FDA is Beginning to Accept New Endpoints and “Real World” Data in Clinical Trials

According to a story from Charcot-Marie-Tooth Disease News, the U.S. Food and Drug Administration (FDA), under the leadership of commissioner Scott Gottlieb, has begun to change clinical trials by accepting…

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The City of Hope Just Received Millions in Funding To Test A New Procedure to Treat Sickle Cell Disease
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The City of Hope Just Received Millions in Funding To Test A New Procedure to Treat Sickle Cell Disease

According to a story from bioportfolio.com, The California Institute for Regenerative Medicine has given $5.74 million to the City of Hope, a private, nonprofit clinical research center that is primarily…

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