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Last week, 100,000 Americans with sickle cell disease and millions more around the world got encouraging news. Investors gave a vote of confidence, in the form of a $120 million IPO, to Global Blood Therapeutics (NASDAQ: GBT), a four-year-old biotech working on a pill that could become the biggest medical advance ever for the disease.
That pill, which just began its first human trial in January, is a once-a-day medicine that a patient would have to take for life. If approved, the drug would be the only sickle cell treatment to potentially halt the effects of the disease. The lone drug currently on the market for the disease only addresses some of its symptoms.
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This platform is made possible through a partnership with the Sickle Cell Disease Association of America, Inc. (SCDAA) and its member organizations. SCDAA's mission is to advocate for people affected by sickle cell conditions and empower community-based organizations to maximize quality of life and raise public consciousness while advancing the search for a universal cure.