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
La Jolla Pharmaceutical company announces initiation of pivotal clinical study of LJPC-401 in patients with beta thalassemia
La Jolla Pharmaceutical company today announced the initiation of a pivotal clinical study of LJPC‑401 (synthetic human hepcidin) in patients with transfusion-dependent beta thalassemia who, despite chelation therapy, have cardiac iron levels above normal. A high level of cardiac iron puts patients at risk of cardiac complications such as heart failure and sudden death.
LJ401-BT01 is a pivotal, multinational, multicenter, randomized, controlled study that is designed to enroll approximately 100 patients across 9 countries, including the United States. Patients will be randomized 1:1 to receive either: (i) weekly subcutaneous injections of LJPC‑401, while continuing standard-of-care chelation therapy (LJPC‑401 treatment arm); or (ii) a continuation of standard-of-care chelation therapy only (observation arm).


Related Content
-
videos & visualsWhat Should Teens Ask Their Doctors About Hydroxyureahttps://www.youtube.com/watch?v=VZn15uXQ...
-
education & researchAcute Care Utilization at End of Life in Sickle Cell Disease: Highlighting the Need for a Palliative ApproachBackground: People with sickle cell dis...
-
videos & visualsThe neglected and forgotten Sickle Cell Disease – A silent mass killer in Kenyahttps://www.youtube.com/watch?time_conti...
-
news & eventsBluebird bio acquires Durham, NC, manufacturing site for lentiviral vector productionbluebird bio said today it has acquired ...
-
news & eventsGBT Receives FDA Breakthrough Therapy Designation for Voxelotor for Treatment of Sickle Cell Disease (SCD)Global Blood Therapeutics, Inc. (GBT) to...
-
education & researchCollege Students Living with a Chronic ConditionThe term "chronic conditions" may apply ...
-
news & eventsHow One Child’s Sickle Cell Mutation Helped Protect the World From MalariaThousands of years ago, a special child ...
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.