Manuela B.
- Country
- Italy
- Department
- S.S.D. Microcitemia e delle Anemie Congenite
Research Overview
- Evaluation of the Efficacy, Safety, and Tolerability of Subcutaneous REGN7999 in Adults with Iron Overload Due to Non-Transfusion Dependent Beta-Thalassemia
- Efficacy and Safety Evaluation of Oral Decitabine-Tetrahydrouridine in Hydroxyurea-Non-Eligible Sickle Cell Disease Patients
- Dose-Escalation and Dose-Finding Study of (S)-4,5-Dihydro-2-[2-Hydroxy-4-(3,6-Dioxaheptyloxy)phenyl]-4-Methyl-4-Thiazolecarboxylic Acid in Transfusion-Dependent α- or β-Thalassemia
Data & Insights
Has run more than one trial at 50 of 98 partner sites.
Specializations
Thalassemia Research
The investigator specializes in both transfusion-dependent and non-transfusion-dependent thalassemia, with particular focus on treating alpha and beta thalassemia variants. Their research encompasses innovative therapeutic approaches for managing thalassemia-related complications.
- Alpha-thalassemia treatment studies
- Beta-thalassemia management approaches
- Transfusion dependency interventions
The work extends to pediatric and adult populations, investigating novel compounds for improving anemia outcomes.
Sickle Cell Disease Studies
The investigator conducts research in sickle cell disease treatment, focusing on addressing both anemia and pain crises. Their work includes evaluation of oral therapeutics and novel treatment approaches for patients who cannot use conventional treatments.
- Anemia management in SCD
- Pain crisis intervention
- Alternative therapeutic options
Research emphasis includes developing treatments for patients with limited therapeutic options.
Rare Blood Disorders
The investigator's expertise extends to rare conditions including Erythropoietic Protoporphyria (EPP) and X-Linked Protoporphyria (XLP). Their research includes long-term safety studies and therapeutic interventions for managing sun exposure sensitivity.
- EPP treatment development
- Iron overload management
- Long-term therapeutic monitoring
The research focuses on improving quality of life for patients with these rare conditions.
Related Investigators
Researchers in similar fields or departments
Want to know more about this investigator?
We can share extended profile information and contact details.
Is something out of date?
Tell us what needs correcting and we'll update this profile.