person
Nicola B. P.
- Country
- Italy
- Department
- Unità di Terapie Innovative per Malattie Genetiche e Metaboliche
Research Overview
assignment
3
Clinical Trials ·
- Phase I/II Open-Label Study of GNT0003 for Safety and Efficacy in Severe Crigler-Najjar Syndrome Requiring Phototherapy
- Phase 3 Randomized Double-Blind Placebo-Controlled Trial of Pariglasgene Brecaparvovec in Glycogen Storage Disease Type Ia Patients
- Phase 3 Randomized Double-blind Placebo-controlled Study of Avalotcagene Ontaparvovec Gene Transfer in Late-onset Ornithine Transcarbamylase Deficiency
location_city
1
Research Sites
Data & Insights
medical_services
Primary Speciality
Main therapeutic area
handshake
Return Partnerships
Recurring site collaborations
2
Sites with multiple studies
Has run more than one trial at 2 of 18 partner sites.
Specializations
Genetic Metabolic Disorders
This investigator specializes in the treatment of rare genetic metabolic disorders, focusing on innovative therapeutic approaches for conditions affecting essential metabolic pathways.
- Crigler-Najjar syndrome management
- Ornithine Transcarbamylase (OTC) deficiency treatment
- Glycogen Storage Disease Type Ia therapy
Gene Therapy Applications
The investigator focuses on developing gene therapy solutions for inherited metabolic conditions, particularly those affecting liver function and metabolic pathways.
- Single-dose therapeutic interventions
- Intravenous gene transfer techniques
- Vector-mediated gene delivery systems
Metabolic Disease Management
Expertise in treating complex metabolic disorders with focus on enzyme deficiency conditions and glucose metabolism disorders.
- Phototherapy requirement assessment
- Ammonia level management
- Glucose control optimization
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