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,…
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…
The agency in Guangzhou, China, that arranged three-year-old Shelby’s adoption by a family in the US disclosed to Michelle and Adam Campbell that Shelby had beta thalassemia disorder. Yet, that…
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…
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…
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…
According to a story from Vice World News, the Solanki family, who are residents of Ahmedabad, the largest city in India's western state of Gujarat, recently took action to save…
According to a story from BioPortfolio, the gene therapy company bluebird bio, Inc. recently announced that its gene therapy ZYNTEGLO™ has earned conditional marketing authorization in the European Union from…
According to a story from Market Screener, the biopharmaceutical company Orchard Therapeutics recently announced that the results of a recent clinical trial testing the company' investigational product OTL-300 were encouraging.…
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…
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…
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…
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…
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