According to a press release from Street Insider, the biopharmaceutical company Fulcrum Therapeutics, Inc., has recently announced positive interim findings from its ongoing phase I clinical trial, which consists of…
Unfortunately, there are a great deal of inequities within the healthcare system. Health inequity occurs when groups are treated differently, offered different or worse care, or have a lower…
Medscape reported that when Victoria Gray was three months old, her family was told to keep her close: her prognosis wasn't looking good. She had been rushed to the ER…
In a news release from May 1, 2021, Chiesi Global Rare Diseases, a business unit of the pharmaceutical company the Chiesi Group, shared its FDA acceptance of FERRIPROX (deferiprone). Altogether,…
Welcome to the Rare Classroom, a new series from Patient Worthy. Rare Classroom is designed for the curious reader who wants to get informed on some of the rarest, most…
Written by Gina Glass Like any parent of a child with a rare disease, my world was turned upside down when my daughter, Gia, was diagnosed with sickle cell disease…
As reported in PR NewsWire; in an effort to help the entirety of the sickle cell disease community, Emmaus Life Sciences, Inc has announced a program that will provide their…
Early last week, Agios Pharmaceuticals ("Agios") announced that its treatment Mitapivat received Orphan Drug designation. This orally administered therapy is a pyruvate kinase R (PKR) activator. As Agios has…
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…
Global Blood Therapeutics (GBT) will submit their Marketing Authorization Application (MAA) for Oxbryta to the EMA by 2021, says a recent BioSpace press release. Ultimately, the MAA will seek regulatory…
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…
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…
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,…
As reported in Biospace, Fulcrum Therapeutics has announced it will be submitting an application to the FDA for FTX-6058 as an Investigational New Drug (IND) by the end of 2020…
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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…
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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…
There is no known cure for sickle cell disease. Treatment is symptomatic and meant to prolong life. While a blood and bone marrow transplant is an option, not everyone…
Grajevis Bakatunkanda lived in the Democratic Republic of the Congo, where he received treatment for malaria after experiencing weekly pain crisis. The treatment he received did not work, as…
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…
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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