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
-
Acute Chest Syndrome in Children with Sickle Cell DiseaseAcute chest syndrome (ACS) is a frequent...
-
New Method of Creating Healthy Stem Cells Could Potentially Improve Treatment of Sickle Cell AnemiaResearchers at New York’s Weill Cornel...
-
Association of Matched Sibling Donor Hematopoietic Stem Cell Transplantation With Transcranial Doppler Velocities in...Importance: In children with sickle cel...
-
FDA Approves Glutamine Powder for Sickle Cell DiseaseThe U.S. Food and Drug Administration ap...
-
Siklos (hydroxyurea) now available through ProCare Pharmacy Care’s Siklos At Home programMedunik USA announced today that...
-
Hospital Playroom Transformed Into Romantic Restaurant For Parents Of Kids With Bone Marrow TransplantsIt was Valentine’s Day with a lot of h...
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.