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
-
Emmaus, a leader in sickle cell disease treatment, signs agreement with McKesson to expand distribution network for ...Emmaus Life Sciences, Inc., anno...
-
Sickle Cell Foundation Nigeria, Rhieos Develop 1st Multi-SCD Registry in NigeriaIn a bid to improve quality of care for ...
-
Ketamine Infusion for Pain Control in Acute Pediatric Sickle Cell Painful CrisesOBJECTIVES: Sickle cell disease is the ...
-
High mortality among children with sickle cell anemia and overt stroke who discontinue blood transfusion after trans...BACKGROUND: Chronic blood transfusion is...
-
Imara Reports Favorable Preclinical and Phase 1 Data on IMR-687 in Sickle Cell DiseaseImara Inc. today announced it will repor...
-
Crizanlizumab lowers pain crises in at-risk sickle cell patients, ad-hoc trial data showNovartis’ investigational th...
-
FOA TA Webinar: Sickle Cell Disease Clinical Data Collection Platformhttps://www.youtube.com/watch?v=8afutDfe...
Don’t forget to join the oneSCDvoice community!
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
Don’t forget to join the oneSCDvoice community!
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.
