Àngels G. C.
- Country
- Spain
- Department
- Endocrinology
Research Overview
- Long-Term Safety Follow-Up of AAV‑RH79‑OTC Gene Therapy (ECUR‑506D) in Patients with Ornithine Transcarbamylase Deficiency
- Evaluation of Radiprodil's Safety, Tolerability, and Pharmacokinetics in Pediatric GRIN-Related Disorder: A Multicenter Study
- A Phase 1/2, Global, Open-Label, Extension Study to Evaluate the Long-Term Safety and Clinical Activity of mRNA-3705 in Participants Previously Enrolled in Other Clinical Studies of mRNA-3705.
Data & Insights
Has run more than one trial at 12 of 37 partner sites.
Specializations
Metabolic Disorders
The investigator specializes in treating rare metabolic conditions, with particular focus on Propionic Acidemia and Methylmalonic Acidemia. Their research explores innovative therapeutic approaches for managing these inherited metabolic disorders.
- Metabolic Disease Events Management
- Intravenous Therapeutic Applications
- Enzyme Deficiency Treatment
Their work addresses critical metabolic pathway disruptions and their clinical manifestations.
Neurological Conditions
The investigator conducts research in X-linked Adrenoleukodystrophy, particularly its cerebral form in pediatric patients. Their work involves monitoring disease progression through advanced imaging techniques.
- Pediatric Neurological Disorders
- MRI-based Disease Monitoring
- Pre-transplant Therapeutic Interventions
Their expertise extends to evaluating therapeutic interventions for progressive neurological conditions.
Fatty Acid Metabolism
The investigator focuses on Long-chain Fatty Acid Oxidation Disorders, examining therapeutic approaches using specialized triglycerides. Their research evaluates clinical outcomes in pediatric patients with metabolic disorders.
- Lipid Metabolism Disorders
- Clinical Event Prevention
- Nutritional Intervention Studies
Their research encompasses comparative studies of different therapeutic approaches in metabolic disorders.
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