A recent article in the journal Nature elaborated on the work of Dr. Ted Love who left early retirement in 2013 to devote his talents and experience to the development…
Three doctors recently got together to discuss how they manage care for their sickle cell disease (SCD) patients. As a part of this conversation, they discussed how they combat systemic…
According to The Guardian, England’s National Health Service (NHS) has announced that a new therapeutic option will be available for patients with sickle cell disease (SCD). This treatment, called crizanlizumab,…
According to a story from MSN, Busola Omiyale's daughter Toriayooluwa was only nine months old when she could tell that something wasn't right. The girl had sickle cell disease, a…
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…
Fulcrum Therapeutics recently announced the interim results from their Phase 1 trial of FTX-6058 in a press release. This selective small-molecule inhibitor of EED is in development for the treatment…
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…
Currently, the European Hematology Association (EHA) is holding its 2021 Virtual Congress through June 17, 2021. During the event, researchers are expounding on new insights and research within the hematological…
Have you ever heard of base editing? This relatively new technology within the field of genome editing offers the potential opportunity to treat a variety of genetic conditions. According to…
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…
CRISPR has seen a number of developments recently; 2020 was a good year for gene therapy. Medical professionals are excited by the improvements made and motivated to make further discoveries.…
By: Malika Abrams I was eight years old when I found out that I was sick. After spending a beautiful summer day at the community pool with my parents, brothers,…
While gene therapy is a promising field, there are some associated risks: unwanted immune reactions, infections, or whether the therapy could lead to the development of other conditions, like cancer.…
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…
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…
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