Clinical TrialsInformation and insight on the latest clinical trials
- Epstein-Barr Virus Associated Post-transplant Lymphoproliferative Disorder (EBV+ PTLD)
Mohamad Mohty, MD, PhD, discusses EBV+ PTLD is an aggressive lymphoma that occurs following bone marrow transplant (also known as hematopoietic stem cell transplant, or HCT) or solid organ transplant (SOT).
- The Current State of Rett Syndrome
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 the current state of Rett Syndrome.
- 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.
- The Role of MEK Inhibitors in NF1
Annette Bakker, PhD, President and Chief Scientific Officer, of the Children’s Tumor Foundation discusses neurofibromatosis, also known as NF, is a genetic disorder that affects 1 in every 3,000 people. There are three types of NF: NF1, NF2, and schwannomatosis.
- Hemophilia: Updated Positive Interim Data from Phase 2/3 Study of Marzeptacog Alfa
Dr. Howard Levy, Chief Medical Officer of Catalyst Biosciences discusses hemophilia and his company’s post at the meeting: Updated Positive Interim Data from Its Phase 2/3 Study of Marzeptacog Alfa (Activated) in Individuals with Hemophilia A or B with Inhibitors.
- Myelodysplastic Syndromes Overview and Personalized Prediction Model for Patients
Aziz Nazha, MD of Cleveland Clinic sat down with CheckRare to provide an overview of myelodysplastic syndromes (MDS), a group of blood disorders characterized by abnormal development of blood cells within the bone marrow.
- Phase 3 Trial Initiated to Evaluate Treatment for Dermatomyositis
A Phase 3 trial to investigate lenabasum as a potential treatment for dermatomyositis, a rare and often fatal multisystem inflammatory autoimmune disease affecting muscle and skin, have been initiated by Corbus Pharmaceuticals.
- Venclexta Data Positive for AML
Rod Humerickhouse, MD, PhD, asset strategy leader at AbbVie discusses some of the Venclexta (Venetoclax) data presented at this year’s 60th American Society of Hematology’s 2018 annual meeting in San Diego.
- 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.
- 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.