A specialist from Oman, located at the crossroads of Africa and Asia, explains that his nation serves as a microcosm when it ...
Zydus Lifesciences announced a significant development for beta-thalassemia patients. The US Food and Drug Administration has ...
Vertex Pharmaceuticals reported on Monday that it beat Wall Street estimates for third-quarter results, driven by strong demand for its cystic fibrosis treatments and early contributions from newer ...
said Joanne Lager, MD, Chief Medical Officer. "The presentation highlights the growing demand for ZYNTEGLO, the first FDA-approved gene therapy for eligible patients with transfusion-dependent ...
A new research project at the Cyprus Institute of Neurology and Genetics (CING) aims to develop a one-time, curative therapy for beta-thalassaemia, the most common inherited blood disorder in Cyprus.
DelveInsight’s “Beta Thalassemia Pipeline Insight 2025” report provides comprehensive insights about 22+ companies and 22+ pipeline drugs in the Beta Thalassemia pipeline landscape. It covers the Beta ...
Vertex Pharmaceuticals (Nasdaq: VRTX) announced today a reimbursement agreement with the Italian Medicines Agency (AIFA) for eligible transfusion-dependent beta thalassemia (TDT) and severe sickle ...
Exagamglogene autotemcel (exa-cel), a CRISPR-Cas9 gene-edited cell therapy, led to sustained improvements in health-related quality of life (HRQOL) for both adults and adolescents with ...
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Agios drops as FDA delays review of thalassemia drug
Agios Pharmaceuticals (NASDAQ:AGIO) fell ~17% in the premarket on Thursday after the U.S. FDA issued a three-month extension to the review period for the company’s lead asset, Pyrukynd, targeted at ...
On Thursday, the U.S. Food and Drug Administration (FDA) extended the Prescription Drug User Fee Act (PDUFA) goal date for Agios Pharmaceuticals, Inc.’s (NASDAQ:AGIO) supplemental New Drug Application ...
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