Acknowledgement: This article was shared with Patient Worthy by our friends at Stork Genetics. To see the original article, please click here. For more information about women affected by X-linked…
The U.S. Medicaid program has taken a pioneering step to ensure that new gene therapies for sickle cell disease (SCD) are accessible to the millions of Americans who rely on…
Sickle Cell, an inherited disease, involves crescent shaped red blood cells that are the “vehicles” containing hemoglobin, a protein responsible for carrying oxygen throughout the body. For almost a year…
Almost 100,000 people in the United States have been diagnosed with sickle cell disease (SCD). About one million people worldwide have the disease. Patients are at risk of death in…
On December 13, 1996, Health Canada issued a policy statement called Priority Review of Drug Submissions which: provided for the "fast-tracking" of eligible New Drug Submissions (NDS) and Supplemental New…
Exa-cel Gene-Edited Therapy Vertex Pharmaceutical recently announced that Health Canada has accepted its New Drug Submission and granted Priority Review for Exa-cel, a gene-edited cell therapy, for the treatment of…
People living with blood disorders like sickle cell disease or thalassemia will now have access to a new blood test that will reduce transfusion side effects. England’s National Health Service…
According to a story from Healio, a recent study has found that use of hydroxyurea in alignment with treatment guidelines has the potential to bring down symptoms of cerebrovascular disease…
CRISPR was first described ten years ago as a viral mechanism for defense, taken from bacteria, which could be transformed into genetic scissors and accurately alter DNA. About Casgevy CRISPR…
By Sharon King, Advocacy and Community Engagement Manager with Aldevron Living with the excruciating pain, debilitating fatigue, and other symptoms of sickle cell disease is challenging. Powerful painkillers and blood…
Throughout her seventeen years alive, Daisha Felps has learned to face obstacles with resilience and strength. And obstacles come around more than she would like. You see, Felps has sickle…
The month of September is recognized as National Sickle Cell Awareness Month in the US. This is a time to spread awareness about sickle cell disease among the general public…
The later part of 2023 will see decisions on drugs in four areas such as advanced melanoma, sickle cell disease, Alzheimer’s disease, and depression. BioSpace shines the spotlight on…
According to a story from BioPharma Reporter, the company bluebird bio has recently submitted its Biologics License Application (BLA) to the US Food and Drug Administration (FDA). This application is…
Welcome to Study of the Week from Patient Worthy. In this segment, we select a study we posted about from the previous week that we think is of particular interest…
In 2014, the National Heart, Lung, and Blood Institute released updated clinical guidelines in reference to the care of children with sickle cell anemia. The updated guidelines recommended the use…
As bluebird bio ("bluebird") waited on FDA feedback regarding the manufacturing process for lovotibeglogene autotemcel (lovo-cel) genetherapy, the company disclosed that it would most likely miss its Q1 submission…
A therapy is granted Orphan Drug designation if the FDA believes that this therapy will treat, prevent, or diagnose rare conditions. Rare conditions, in the United States, are those affecting…
On the field, Running Back (RB) Tevin Coleman has done some amazing things. He has played in 94 games, gained 3,319 rushing yards, and scored 25 touchdowns. Off the…
The 64th ASH Annual Meeting held on December 10, 2022, focused on various approaches for hematologic diseases with the goal of improving quality care and outcomes. Dr. Stephanie Lee, of…
One year ago, in December 2021, clinical studies of lovo-cel for patients who were eighteen or younger were put on partial hold by the FDA. At the time, bluebird…
Before you read, don't forget to check out Part 1 of our interview! In Part 1, we discuss Ronya's story, genetic counseling, and why she chose to launch DNA in Color.…
Welcome to Study of the Week from Patient Worthy. In this segment, we select a study we posted about from the previous week that we think is of particular interest…
In the past, there have been a small number of studies exploring the connection between sickle cell disease (SCD) and craniosynostosis. More recently, researchers sought to understand whether there was…
Happy Friday! A tropical storm system will be traveling up through the mid-Atlantic this weekend, bringing wind and rain. Hunker down! This week, we have stories on Duke University being…
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