Clinical TrialsInformation and insight on the latest clinical trials
- X-linked Severe Combined Immunodeficiency Overview
Manuel Litchman, MD, President, Chief Executive Officer at Mustang Bio discusses his company’s X-linked severe combined immunodeficiency (SCID) program.
- Rising to the Challenge: Developing New Medicines for Rare Disease
The process for researching and developing new medicines is notoriously time-consuming, complicated and costly – and even more so when it comes to therapies for rare diseases.
- FDA Approves Cablivi for Acquired Thrombotic Thrombocytopenic Purpura
The U.S. Food and Drug Administration (FDA) approved Cablivi (caplacizumab-yhdp) injection, the first therapy specifically indicated, in combination with plasma exchange and immunosuppressive therapy, for the treatment of adult patients with acquired thrombotic thrombocytopenic purpura (aTTP), a rare and life-threatening disorder that causes blood clotting.
- Using Genomics to Better Understand Rare Diseases
Daniel MacArthur, PhD is an Institute Member at the Broad Institute of MIT and Harvard, and co-lead of the Broad’s Center for Mendelian Genomics and Rare Genomes Project.
- Sickle Cell Disease: Overview and Emerging Stem Cell Transplant Therapy
Mitchell Cairo, MD, Chief of Pediatric Hematology, Oncology and Stem Cell Transplantation at New York Medical College discusses his study at the 2018 American Society of Hematology (ASH) Annual Meeting in San Diego focusing on Sickle cell disease, a group of inherited red blood cells disorders.
- FDA Approves Imbruvica-Gazyva Combo for Treatment-Naive Chronic Lymphocytic Leukemia
The FDA approved ibrutinib for use in combination with obinutuzumab for treatment-naive patients with chronic lymphocytic leukemia (CLL) or small lymphocytic lymphoma. This is the first nonchemotherapy regimen indicated for this patient population.
- Hereditary TTR Amyloidosis and the NEURO-TTR Study
Morie Gertz, MD, Hematologist and Chair of Internal Medicine at Mayo Clinic discussed the current unmet needs in the treatment of hereditary TTR amyloidosis, antisense technology, inotersen, and the NEURO-TTR study.
- Positive Top-Line Results From Phase 3 Study Of Ultomiris in Atypical Hemolytic Uremic Syndrome
Alexion announced positive top line results in the Phase 3 study of Ultomiris (ravulizumab-cwvz), the long-acting C5 complement inhibitor, met its primary objective in complement inhibitor-naïve patients with atypical hemolytic uremic syndrome (aHUS).
- Cholestatic Liver Diseases: Emerging Treatment Options
Ron Cooper, President and CEO of Albireo, provides and overview of Cholestatic liver diseases. Cholestasis is a chronic condition resulting from an impairment of the biliary system, that provokes a decrease or interruption of bile secretion from the liver to the intestine, leading to the degeneration of liver tissues, chronic inflammation and the formation of scar tissue (fibrosis).
- FDA Approves Cabozantinib for Advanced Hepatocellular Carcinoma
Despite a government shutdown, the FDA approved cabozantinib (Cabometyx) as a treatment for patients with hepatocellular carcinoma (HCC) who previously received sorafenib (Nexavar).