Yusara Ahmed was prepared to take action when she learned that her son Yusuf was diagnosed with beta thalassemia, an uncommon blood disorder. Yusara's sister also lived with the disease,…
Rare Community Profiles is a Patient Worthy article series of long-form interviews featuring various stakeholders in the rare disease community, such as patients, their families, advocates, scientists, and more.…
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…
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…
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…
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…
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,…
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On February 3, 2020, twenty-seven newly installed flags of the European Union stood in the gleaming lobby of the new European Medicines Agency (EMA) headquarters in Amsterdam. The Charcot-Marie-Tooth…
According to a story from globenewswire.com, the drug developer Agios Pharmaceuticals, Inc. has recently released a preliminary analysis of phase 2 trial findings that have served to establish proof-of-concept for…
La Jolla Pharmaceutical recently announced a new clinical trial targeting thalassemia. The trial, described as pivotal, investigates a drug referred to as LJPC-401. The treatment is intended to help patients…
A new case study suggests that the blood disorder, thalassemia, may be present in people with Gaucher disease. Both diagnosis and treatment become more complicated if this hypothesis proves true.…
October 21, 2017 Remember this deadline if you have a alpha or beta thalassemia, or another significant form of thalassemia, and are seeking an advanced degree in the US or abroad…
Sickle cell disease, hemophilia, and thalassemia are a few of the inherited blood disorders. Dr. Waveney Charles is a hematologist, or specialist in blood-related conditions. She believes that the number…
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