Clinical TrialsInformation and insight on the latest clinical trials
- iPATH Study: Key Biomarkers That Affect Bleeding Patterns, Response to Treatment for Hemophilia A
Peter Turecek, Global Medical Affairs, Shire, discusses the Irish Personalised Approach to the Treatment of Haemophilia (iPATH) study, an ongoing collaboration investigating new personalized treatment approaches by tailoring care based on the needs of individual patients.
- New Treatment Option for Primary Hemophagocytic Lymphohistiocytosis
Michael Jordan, MD, a physician-scientist in the division of Bone Marrow Transplantation and Immune Deficiency at Cincinnati Children’s Hospital Medical Center HLH Center of Excellence, discusses primary hemophagocytic lymphohistiocytosis (HLH) and the phase 2/3 clinical trial presented at the American Society of Hematology (ASH) Annual Meeting in San Diego.
- ASH ’18: Management of Hematologic Diseases With Cellular and Immunotherapies
Mohamad Mohty, MD, discusses the management of hematologic diseases with cellular and immune therapies such as Chimeric antigen receptor (CAR) T cells, which have recently emerged as a novel therapeutic approach, yielding complete responses even in heavily pretreated patients with leukemia, lymphoma, and myeloma.
- Luspatercept Successful in Late-Stage Beta Thalassemia Study
A Phase 3 clinical trial evaluating luspatercept in adults with beta-thalassemia-associated anemia met the primary endpoint. The results were presented at the American Society of Hematology (ASH) in San Diego.
- Krabbe Disease
Melissa P. Wasserstein, MD, Chief of Pediatric Genetic Medicine at the Children’s Hospital at Montefiore, New York City discusses Krabbe disease (also called globoid cell leukodystrophy), a degenerative disorder that affects the nervous system.
- Leber’s Congenital Amaurosis Overview
Daniel de Boer, Founder and CEO of ProQR, discussuses Leber’s congenital amaurosis (LCA), the most common genetic cause of childhood blindness.
- FDA Grants Orphan Drug Designation to Gene Therapy for CLN2 Form Of Batten Disease
The U.S. Food and Drug Administration (FDA) granted Orphan Drug Designation to RGX-181, a one-time treatment candidate for late-infantile neuronal ceroid lipofuscinosis type 2 (CLN2) disease, one of the most common forms of Batten disease caused by mutations in the tripeptidyl peptidase 1 (TPP1) gene.
- Rett Syndrome Natural History Study
Daniel Glaze, Medical Diorector of the Blue Bird Circle Rett Center and a Professor in the Departs of Pediatrics and Neurology at Baylor College of Medicine in Houston discusses the Rett Syndrome Natural History Study.
- Rett Syndrome Research and Clinical Trials
Daniel Glaze, Medical Director of the Blue Bird Circle Rett Center and a Professor in the Departments of Pediatrics and Neurology at Baylor College of Medicine in Houston, discusses ongoing Rett syndrome research and clinical trials, including the ongoing phase 3 STARS trial.
- Sickle Cell Trait Increases Risk for Some Health Conditions
In the United States, an estimated 2.5 to 3 million people have a gene variant associated sickle cell trait (SCT) and most never have related health complications. Researchers have now identified three health conditions for which people with SCT are at increased risk.