Google
Trusted Resources: News & Events
Latest announcements and gatherings
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.
+myBinderRelated Content
-
Effect of crizanlizumab on pain crises in subgroups of patients with sickle cell disease: A SUSTAIN study analysisThe cell adhesion molecule P-selectin pl...
-
Hydroxyurea (hydroxycarbamide) for sickle cell diseaseBACKGROUND: Sickle cell disease (SCD) i...
-
Patients frequently turning to cannabis to treat symptoms of sickle cell diseaseMedical cannabis use is relatively commo...
-
Vaso-Occlusive Pain Linked to Menstruation in Some Patients, Study FindsAcute vaso-occlusive pain is associated ...
-
Global Sickle Cell Disease Treatment Market 2019–2023| Evolving Opportunities with ADDMEDICA and Bristol-Myers Squ...The global sickle cell disease treatment...
-
Phase 3 Trial of Rivipansel in Treating SCD Pain Crisis Fails to Meet Goals, Pfizer AnnouncesThe Phase 3 RESET trial testing the effe...
-
HHS Secretary Alex Azar Touts White House Efforts to Fight Sickle Cell DiseaseBy 2029, Americans with sickle cell dise...
To improve your experience on this site, we use cookies. This includes cookies essential for the basic functioning of our website, cookies for analytics purposes, and cookies enabling us to personalize site content. By clicking on 'Accept' or any content on this site, you agree that cookies can be placed. You may adjust your browser's cookie settings to suit your preferences. More Information
The cookie settings on this website are set to "allow cookies" to give you the best browsing experience possible. If you continue to use this website without changing your cookie settings or you click "Accept" below then you are consenting to this.
Powered by
Support for this site is provided by
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.