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- FDA Approves First Gene Therapy for Young Children with Sickle Cell DiseaseThe U.S. Food and Drug Administration today issued a supplemental approval for Casgevy (exagamglogene autotemcel) for patients aged 2 years and older with either sickle cell disease (SCD) with recu…fda press · 28d ago↗ source
- Merck scraps a Phase 2 Alzheimer's asset from partner NeuphoriaMerck has axed a Phase 2 trial testing an experimental Alzheimer’s medicine, Endpoints News has learned. The company terminated a mid-stage test of the oral drug candidate MK-1167, a Merck spokespe…Endpoints · 28d ago↗ source
- FDA warns Genzyme's Irish site over quality questionsAn FDA warning letter sent late last month to Genzyme's Ireland-based manufacturing site, now owned by Sanofi, raises significant quality- and data-related issues. On data integrity, the warning le…