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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.
education & researchDexmedetomidine as an adjuvant to analgesic strategy during vaso-occlusive episodes in adolescents with sickle cell ...Sickle cell disease (SCD)-patients exper...
videos & visualsCord Blood Transplants: A Sickle Cell Curehttps://www.youtube.com/watch?v=8EaJr3Qp...
news & eventsInvestigational Therapy Altemia Achieves Main Endpoints in Pediatric Sickle Cell TrialSancilio Pharmaceuticals says its invest...
news & eventsbluebird bio Announces Publication of Case Study on First Patient with Severe Sickle Cell Disease Treated with Gene ...Patient treated with LentiGlobinTM drug ...
news & eventsSickle Cell Patient Receives CRISPR Gene TherapyMany human diseases can be traced back t...
education & researchFrom total blood exchange to erythrocytapheresis and back to treat complications of sickle cell diseaseErythrocytapheresis is an important proc...
news & eventsEmmaus Life Sciences Receives Validation From European Medicines Agency on Marketing Authorization Application for S...Emmaus Life Sciences, Inc. announced tod...
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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.