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…
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…
Josephine Bila was born with transfusion dependent beta thalassemia, a rare blood disorder. Recently, Josephine uploaded a video to Youtube in which she talks about her life as a patient,…
A press release from the US Food and Drug Administration (FDA) is announcing the approval of a new treatment for anemia in adult patients with the rare blood disorder beta…
According to a publication from BNN Bloomberg, regulatory authorities in the European Commission recently granted conditional marketing authorization (CMA) to Zynteglo. Zynteglo is a gene therapy designed to treat beta…
According to a story from Stockwatch, the gene therapy company bluebird bio, inc. recently announced that the European Medicine Agency's (EMA) Committee for Medicinal Products for Human Use (CHMP) has…
According to a story from BioPortfolio, the biopharmaceutical company Acceleron Pharma Inc., recently announced the release of results from a Phase 2 study which tested the drug luspatercept as a…
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