Review of Rare Movement Disorders in India and the United States

A recent article published in India’s Pharma Literati cites The World Health Organization as not having endorsed a definition for rare diseases. The European Union defines 'rare' as less than…

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Gene Therapy: The New Frontier for Cancer

  Gene therapy is the treatment of disease by way of the transfer of genetic material into cells. According to a recent article in Labiotech, an EU publication, gene therapy…

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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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Symptom Severity in Rare Disease Varies by Patients: Genetics May be to Blame

Geisinger was founded by Abigail Geisinger over 100 years ago. It now has 13 hospitals and 2 research centers. They just released a new study examining the severity of symptoms…

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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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CRISPR-Cas9: Scientists and Patients Have Philosophical Differences

  Calls to Ban CRISPR A scientist named He Jiankui recently shocked the scientific world with his announcement that he had created twin “CRISPR babies”. The babies, a result of…

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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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Collaborative Effort to Improve Patient Access to Personalized Medicine

The FDA previously announced their plan to hire 50 new employees specifically to manage the influx of gene therapy applications. Currently, they have approximately 800 applications awaiting review and they…

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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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New Algorithm May Diagnose Cystic Fibrosis Faster than Physicians
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New Algorithm May Diagnose Cystic Fibrosis Faster than Physicians

A diagnosis means everything for a rare disease patient. However, to process 200 patient records manually, it would take the most dedicated physician approximately 40 hours. That's one full work…

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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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