Niamh O.
- Country
- Ireland
- Department
- Haematology
Research Overview
- A phase 3b open-label, multicenter study evaluating physical activity and joint health in previously treated patients ≥12 years of age with severe haemophilia A treated with intravenous recombinant coagulation factor VIII Fc-von Willebrand Factor-XTEN fusion protein (rFVIIIFc-VWF-XTEN; efanesoctocog alfa) for 24 months
- Phase III Randomized Open‑Label Non‑Inferiority Trial of NXT007 Prophylaxis versus Emicizumab in People with Hemophilia A
- Long-term Safety and Efficacy Evaluation of Fitusiran in Hemophilia A or B Patients With or Without Factor VIII or IX Inhibitory Antibodies
Data & Insights
Has run more than one trial at 39 of 108 partner sites.
Specializations
Hematological Disorders
This investigator specializes in treating and researching hemophilia disorders, with particular emphasis on both hemophilia A and B. Their work encompasses comprehensive evaluation of bleeding disorders and their management through various therapeutic approaches.
- Severe and Moderate Hemophilia
- Coagulation Factor Deficiencies
- Bleeding Disorder Management
Their expertise extends to treating patients with inhibitory antibodies to coagulation factors.
Novel Therapeutics
The investigator focuses on evaluating innovative treatment approaches including gene therapy applications and novel prophylactic treatments for bleeding disorders. Their work includes assessment of long-term therapeutic outcomes.
- Advanced Factor Replacement Therapy
- Gene Therapy Development
- Prophylactic Treatment Strategies
Their research encompasses both traditional and emerging therapeutic modalities for hemophilia treatment.
Clinical Outcomes
The investigator specializes in studying patient outcomes with particular attention to joint health assessment and physical activity evaluation in hemophilia patients. Their work includes long-term monitoring of treatment efficacy.
- Physical Activity Assessment
- Joint Status Evaluation
- Long-term Safety Monitoring
Expertise includes ultrasound-based joint assessment and activity level monitoring in patients with bleeding disorders.
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