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
-
Using CRISPR Cas9 to cure sickle cell diseasehttps://www.youtube.com/watch?v=WdkP-RU5...
-
Pneumococcal vaccination coverage among children with sickle cell anemia, sickle cell trait, and normal hemoglobinBackground: Children with sickle cell a...
-
Gene Therapy for Blood DisordersIn the context of intense scrutiny over ...
-
Crizanlizumab 5.0 mg/kg increased the time to first on-treatment sickle cell pain crisis: a subgroup analysis of the...In the 52-week SUSTAIN study, which comp...
-
Siklos (hydroxyurea) now available through ProCare Pharmacy Care’s Siklos At Home programMedunik USA announced today that...
-
Kids With Sickle Cell Anemia are More Sedentary Than Healthy Peers, Study SuggestsChildren and adolescents with sickle cel...
-
Capitol Hill Briefing Held to Encourage Sickle Cell Research and TreatmentEarlier today, a briefing was hosted on ...
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.