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
-
Crizanlizumab for the Prevention of Pain Crises in Sickle Cell Diseasehttps://www.youtube.com/watch?v=CWTW5pDP...
-
Unanticipated consequences identified after implementation of a pediatric emergency department (PED)-based intranasa...Background: The 2014 National Heart Lun...
-
Women with Sickle Cell Disease and Prenatal Care: What to Know During PregnancyPrenatal care is health care you get whi...
-
Matthew Porteus awarded grant for sickle cell anemia workThe California Institute for Regenerativ...
-
Statement from FDA Commissioner on agency’s efforts to advance development of gene therapiesOnce just a theory, gene therapies are...
-
Sickle Cell Disease and Marrow/Stem Cell Donationhttps://www.youtube.com/watch?v=mstThQc7...
-
Gamida Cell Announces $3.5 Million Grant from the Israeli GovernmentGamida Cell, a leader in cellular and im...
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
SomeBodyToTalkTo connects people affected by rare diseases with trusted resources, supportive communities, and opportunities to share their experiences.