Clinical TrialsInformation and insight on the latest clinical trials
- 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.
- American Society of Hematology Conference Coverage
Sickle Cell Disease: Overview and Emerging Stem Cell Transplant Therapy Hemophilia: Updated Positive Interim Data from Phase 2/3 Study of Marzeptacog Alfa Myelodysplastic Syndromes Overview and Personalized Prediction Model for Patients iPATH Study: Ke …
- 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.
- Checkmate 436: Two-Drug Combo Shows Promise for Primary Mediastinal Large B-cell Lymphoma
According to investigators from the CheckMate 436 trial, Nivolumab plus brentuximab vedotin may be a new treatment option for patients with relapsed/refractory primary mediastinal large B-cell lymphoma (PMBCL).
- 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.