According to a story from BioPharma Reporter, the company bluebird bio has recently submitted its Biologics License Application (BLA) to the US Food and Drug Administration (FDA). This application is…
In late May 2023, clinical-stage biopharmaceutical company Sangamo Therapeutics, Inc. (“Sangamo”) shared that its product isaralgagene civaparvovc (ST-920) was granted Fast Track Designation by the FDA. This designation is…
“Three years ago, my lung function was 11% and I was hours from death, then I received the gift of life – which was Trikafta.” These are the words of…
Rare Community Profiles Rare Community Profiles is a new Patient Worthy article series of long-form interviews featuring various stakeholders in the rare disease community, such as patients, their…
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