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Trusted Resources: News & Events
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Sickle cell patient with severe anemia rapidly improves with voxelotor, case study shows
Global Blood Therapeutics (GBT) reported a case study in which a sickle cell disease (SCD) patient was granted compassionate access to the company’s ongoing Phase 3 HOPE study of voxelotor (NCT03036813). The patient had severe, transfusion-refractory anemia and experienced rapid improvements with the therapy.
The U.S. Food and Drug Administration (FDA) developed the option of compassionate access, also called “compassionate use,” to enable patient enrollment in an “impossible scenario,” i.e. when a patient is ineligible for ongoing trials or no ongoing trials exist. With this measure, patients may be able to receive the investigational therapy when appropriate.
videos & visualsHydroxyurea and Sickle Cell Disease: A HRSA EMBRACE Projecthttps://www.youtube.com/watch?v=AyMP3P9E...
education & researchAssociation of Antibiotic Choice With Hospital Length of Stay and Risk Factors for Readmission in Patients With Sick...Study objective: We determine the assoc...
education & researchQ & A Facts on Hydroxyurea for Children With Sickle Cell DiseaseIt is time to rethink hydroxyurea for th...
news & eventsPhase 1 trial to test under-the-skin injection of sevuparin in sickle cell patientsModus Therapeutics is going to launch ...
news & eventsCapitol Hill Briefing Held to Encourage Sickle Cell Research and TreatmentEarlier today, a briefing was hosted on ...
Community CenterSequence Replacement to Cure Sickle Cell DiseaseNext-generation gene editing is already ...
news & eventsPreclinical Data on Proprietary AKT Inhibitor, ARQ 092, Demonstrating Effectiveness in the Treatment of Sickle Cell ...ArQule, Inc. (Nasdaq: ARQL) today announ...
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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.