Sickle Cell Disease: FDA Lifts Partial hold on bluebird’s Trial for Patients Under 18
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…
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…
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…
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…
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…
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…
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…
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…
On December 9, 2021, the National Organization for Rare Disorders (NORD) hosted a webinar program titled "Health Equity and Rare Disorders." This webinar was the first of a three-part series…
CRISPR is a gene-editing technique that has revolutionized the medical world with its approach to making precise changes in DNA. CRISPR found its match in 35-year-old Victoria Gray of…
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…
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…
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…
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,…
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…
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…
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…
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…
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…
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,…
A recent article published in India’s Pharma Literati cites The World Health Organization as not having endorsed a definition for rare diseases. The European Union defines 'rare' as less than…