by Lauren Taylor from In The Cloud Copy Sickle cell disease or SCD is a type of red blood cell disorder in which the normally round red blood cells are…
On December 9th, 2020, a special edition of "Behind the Mystery: Rare and Genetic" will air on Lifetime TV as part of the acclaimed morning program The Balancing Act. It…
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…
By Lauren Thayer from In The Cloud Copy Sickle cell disease (SCD) is group of inherited red blood cell disorders. In a typical, healthy red blood cell, the cell is…
June 19th is recognized as World Sickle Cell Day. This day is set aside to help spread awareness about sickle cell disease and its variants among the general public and…
Unfortunately, many people with sickle cell disease are at an increased risk of experiencing central nervous system complications that can reduce quality of life. But more importantly, complications like stroke,…
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 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…
In a post from MD Magazine, a video from the HCPLive Network depicts a conversation between a number of sickle cell anemia experts about different clinical presentations and complications associated…
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…
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…
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…
We've been hearing A LOT about so-called 'gene editing' and 'CRISPR' lately -- and now there's new developments on that front. Scientists have developed a new gene-editing technology called 'prime…
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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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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Popular Science published an article recently about the first time an attempt was made to treat sickle cell anemia using CRISPR technology. The patient, Victoria Gray, age 41, from…
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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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…
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…
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…
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…
According to a story from Nature, Dr. Elliot Vichinsky thinks that almost a third of his patients with sickle cell disease ultimately die from causes that could be prevented. Sickle…
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…
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…
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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