Julian S. N.
- Country
- Spain
- Department
- Pediatric Oncology
Research Overview
- Long-Term Follow-up: Phase I/II clinical study to evaluate the safety and efficacy of the infusion of RP-L102
- Efficacy and Safety Evaluation of Mitapivat in Pediatric Patients with Pyruvate Kinase Deficiency Not Receiving Regular Transfusions: A Phase 3, Multicenter, Randomized, Double-blind, Placebo-Controlled Study
- Efficacy and Safety Evaluation of Subcutaneous Mepolizumab in Pediatric Patients with Hypereosinophilic Syndrome: A 52-Week, Phase 3, Open-Label, Single-Arm Study
Data & Insights
Has run more than one trial at 2 of 34 partner sites.
Specializations
Genetic Disorders
This investigator specializes in treating rare genetic conditions affecting blood cells and metabolism, with particular focus on Pyruvate Kinase Deficiency (PKD) and Fanconi Anemia.
- Inherited metabolic disorders
- Blood cell survival disorders
- Genetic blood disorders
The research involves innovative approaches to treating these rare genetic conditions through cellular therapy.
Gene Therapy
Primary focus on developing autologous CD34+ cell therapies and lentiviral vector-based treatments for various genetic disorders.
- Cell-based gene therapy
- Hematopoietic stem cell treatment
- Genetic modification techniques
The investigator works on long-term safety and efficacy evaluations of gene therapy treatments.
Immunological Disorders
Expertise in treating immune system disorders including Leukocyte Adhesion Deficiency-I (LAD-I) and Hypereosinophilic Syndrome (HES).
- Immune cell disorders
- Pediatric immunology
- Novel therapeutic approaches
The research encompasses both pediatric and adult populations with immune system disorders.
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