The initial CRISPR treatment for sickle cell disease was administered to Victoria Gray in July 2019. Victoria, a resident of Forest, Mississippi, was born with sickle cell and has…
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…
According to an NBC AP news item, early results show that CRISPR, a “tool” that alters DNA permanently in blood cells, has the potential to not only stop the progression…
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…
According to an NBC AP news item, early results show that CRISPR, a “tool” that alters DNA permanently in blood cells, has the potential to not only stop the progression…
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…
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…
The FDA has recently approved of an updated label for Endari, a treatment for sickle cell disease (SCD). This new label will give medical professionals better information, allowing them to…
For years, clinical-stage biopharmaceutical company Fulcrum Therapeutics ("Fulcrum") has worked to develop therapies for patients with rare genetic disorders and diseases. Somewhat recently, their focus was on treating beta thalassemia…
Fulcrum Therapeutics is beginning a Phase 1 trial of FTX-6058, a treatment for sickle cell disease (SCD). Researchers will focus on the tolerability, safety, and pharmacokinetics of the medication. The…
CRISPR Therapeutics, in combination with Vertex Pharmaceuticals, has recently announced that the European Medicines Agency (EMA) has granted the Priority Medicines (PRIME) designation for their sickle cell disease (SCD) treatment,…
A recent study published in the journal Blood Advances has documented that hematopoietic stem cell transplantation (HSCT) is both safe and effective for children with many different kinds of inherited nonmalignant conditions…
Editas Medicine has developed a sickle cell disease (SCD) treatment, and it has recently received the Rare Pediatric Disease designation from the FDA. This means that their therapy, EDIT-301, is…
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 COVID-19 continues to spread across the globe, more and more people are wondering how this virus might affect them. This is especially true for patients with rare diseases,…
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…
Fulcrum Therapeutics has recently announced that it is on track to submit an Investigational New Drug application for their medication, FTX-6058. The application will likely be filed in the second…
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…
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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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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