The United States Food and Drug Administration has awarded Fast Track designation to an investigational drug called PTG-300 for treating chronic anaemia caused by ineffective erythropoiesis in patients who have…
According to a recent press release from Bluebird Bio, the European Medicines Agency has accepted and will review their marketing authorization application (MAA) for the investigational gene therapy LentiGlobin™ as…
According to a story from PR Newswire, the drug developer Protagonist Therapeutics recently announced that its investigational therapy PTG-300 has earned Fast Track designation from the US Food and Drug…
Is there a genetic condition in your family? Are you concerned about the potential for passing this on to your children one day? Do you know if you are a…
According to Bloomberg, there’s a new big fish in the medical technology pond. Crispr Therapeutics AG jumped in value, multiplied by six, and landed at $3 billion. How is that…
The Investigational New Drug Application for the experimental drug CTX001 for treating sickle cell disease has been put on clinical hold, announced Vertex Pharmaceuticals Incorporated and CRISPR Therapeutics. This hold…
According to a story from empr.com, a recent study revealed that gene therapy could allow patients with beta thalessemia to stop having to get regular blood transfusions. The gene therapy…
According to a story from The Motley Fool, The gene therapy development company bluebird bio has been working on a treatment for beta thalassemia, and it has the potential to…
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.…
Hereditary hemochromatosis is a genetic form of a condition in which the body builds up too much iron. To read more about hemochromatosis, click here. La Jolla Pharmaceutical Company has…
How far would you go to eradicate a disease that impacts millions of lives? Would you cross the ethical line if it meant ending a source of suffering forever? Or…
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…
Don’t eat that! Eat this instead. Some diets recommend low fat. Some endorse high protein. Some forbid carbohydrates. Some praise those same carbohydrates. Some limit fiber. It’s so confusing figuring…
β-thalassemia major is a rare blood condition that necessitates a lifetime of blood transfusions. Despite advancements in treatments, the perfect cure has yet to be found. Bluebird Bio, Inc. is…
Celgene Corporation, partnered with Acceleron Pharma Inc., announced at the beginning of this month that target enrollment of myelodysplastic syndromes (MDS) and beta thalassemia patients for the MEDALIST and BELIEVE…
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Most of us know that not having enough iron in the blood results in disorders like anemia, fatigue, low energy… I first became aware of “iron-poor blood,” or anemia, when…
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