DISCLAIMER
The information and materials accessed through or made available for use on any of our Sites, including, any information about diseases, conditions, treatments, or medicines, are for informational purposes only. The Content is not intended to be and is not a substitute for professional medical advice, diagnosis, or treatment, and your participation on our Sites does not create a healthcare professional-patient relationship. You should consult a doctor or other qualified health care professional regarding any questions you have about your health or before making any decisions related to your health or wellness. Call your doctor or 911 immediately if you think you may have a medical emergency.compose your message
message sent
email sent successfully
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.
Related Content
-
education & researchFrom total blood exchange to erythrocytapheresis and back to treat complications of sickle cell diseaseErythrocytapheresis is an important proc...
-
education & researchThe role of nutrition in the pathophysiology and management of sickle cell disease among children: A review of liter...Sickle cell disease (SCD) is one of the ...
-
news & eventsCTX001 Earns FDA’s Fast Track Status for Treating Sickle Cell DiseaseThe U.S. Food and Drug Administration (F...
-
news & eventsADDMEDICA receives FDA-approval for orphan drug Siklos®, first and sole hydroxyurea-based treatment for paediatric ...ADDMEDICA has announced that the...
-
news & eventsASH launches sickle cell disease clinical trials network to accelerate therapy developmentASH launched a clinical trials network d...
-
news & eventsGamida Cell to Present Data from NiCord® Programs at the 2018 BMT Tandem MeetingsGamida Cell, a leading cellular and immu...
-
people & placesThe Sickle Cell Transplant Advocacy & Research Alliance (STAR)The Sickle Cell Transplant Advocacy &...
send a message
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.
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.