Google
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).
+myBinderRelated Content
-
Comparing Sickle Cell Disease Acute Pain Management in the Pediatric and Adult Emergency DepartmentBackground: Vasoocclusive pain leading ...
-
The use of cannabis by patients with sickle cell disease increased the frequency of hospitalization due to vaso-occl...Introduction: The objective of this stu...
-
Novel Sickle Cell Drug Causes Radical Results in End of Life PatientsThis week the FDA approved a novel treat...
-
Blood Transfusions: What You Need to Know and Do (Part 2: Blood Matching)https://www.youtube.com/watch?v=bs4bRApy...
-
Global Blood Therapeutics Announces Completion of Safety Review by Independent Data and Safety Monitoring Board (DSM...Global Blood Therapeutics, Inc. (GBT) (N...
-
Could Gene Therapy Cure Sickle Cell Anemia?Nearly 20 years ago, scientists stunned ...
-
Vitamin D Supplements May Reduce Pain-related Emergency Room Visits in Children With SCDTaking vitamin D supplements may lower t...
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.