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…
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…
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…
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…
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…
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…
Wunmi Bakare described her illness to Essence as a continuous tug-of-war. Balancing sickle cell disease with a nine to five job and trying to stay the course. Then there…
Did you know that September is National Sickle Cell Awareness Month? During this month, various stakeholders come together to share the stories of those affected by sickle cell disease,…
The 5th Annual Las Vegas Sickle Cell Walk September 24, 2022 Dreamsickle Kids Foundation 5th Annual Las Vegas Sickle Cell Walk, this event is the only fundraiser we have to…
For those living with sickle cell disease (SCD), treatment and management can sometimes be difficult. Currently, there is no cure for SCD. Patients may manage their condition using antibiotics,…
Global Blood Therapeutics (GBT) has just announced that their therapeutics called GBT021601 (GBT601) and inclacumab for sickle cell disease (SCD) have received both Rare Pediatric Disease Designation and Orphan Drug…
June 19, 2022 will be recognized as World Sickle Cell Day. This international event is intended to help spread awareness about sickle cell disease among the general public and in…
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…
Prednisone is one of the more common corticosteroids prescribed to individuals with lupus to help reduce inflammation and decrease symptom burden. Corticosteroids are also prescribed for a number of other…
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…
Many patients with sickle cell disease (SCD) experience “pain crises,” or periodic episodes of extreme pain. These pain crises occur when sickle-shaped red blood cells block the flow of blood…
Written By: Teonna Woolford As a child, I never saw myself as anything but “normal.” I loved going to the playground with my friends and jumping rope. My parents encouraged…
Rare Disease Week 2022 Rare Disease Week is the week that leads up to Rare Disease Day, which is recognized on the last day of February. This year, we are…
Victoria Gray’s recent interview with NPR confirmed that her genetically modified blood cells have truly transformed her life. Victoria volunteered in the first-ever attempt in the U.S. to use CRISPR,…
Clinical trials can be extremely beneficial in learning more about various diseases, as well as the safety, efficacy, and tolerability of potential treatments. According to a news release from…
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…
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 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…
Sign Up With a Patient Worthy Account and Share Your Rare Story
- OR -
Make a difference, share your experiences and get paid. Opt-in and join Patient Worthy's panel for paid opportunities such as surveys, market research, patient advisory panels and more.
- OR -
Make a difference, share your experiences and get paid. Opt-in and join Patient Worthy's panel for paid opportunities such as surveys, market research, patient advisory panels and more.
