María Luz C. P.
- Country
- Spain
- Department
- Unit for Diagnosis and Treatment of Metabolic Diseases
Research Overview
- A Long-Term Follow-up Study to Evaluate the Safety and Efficacy of Adeno-Associated Virus (AAV) Serotype 8 (AAV8)-Mediated Gene Transfer of Human Ornithine Transcarbamylase (OTC) in Adults with Late-Onset OTC Deficiency
- 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.
- A Global, Phase 1/2 Study to Evaluate the Safety, Tolerability, Pharmacodynamics, and Pharmacokinetics of mRNA-3705 in Participants with Isolated Methylmalonic Acidemia Due to Methylmalonyl-CoA Mutase Deficiency
Data & Insights
Has run more than one trial at 13 of 32 partner sites.
Specializations
Metabolic Disorders
Specializes in treating rare metabolic conditions, particularly focusing on Ornithine Transcarbamylase (OTC) deficiency and Glycogen Storage Disease Type Ia. The investigator studies innovative gene transfer approaches for these inherited metabolic disorders.
- OTC Deficiency Management
- Glycogen Storage Disease Treatment
- Long-chain Fatty Acid Oxidation Disorders
Explores therapeutic approaches using gene transfer technology to address enzyme deficiencies.
Neonatal Medicine
Conducts research in critical neonatal conditions, with particular emphasis on bronchopulmonary dysplasia and hypoxic-ischemic encephalopathy in premature infants.
- Premature Infant Care
- Neonatal Brain Protection
- Respiratory Support Interventions
Investigates protective therapies for preventing lung injury and improving neurological outcomes in newborns.
Genetic Disorders
Investigates treatments for various genetic conditions including Fabry Disease and Mucopolysaccharidosis Type IIIA. Focuses on developing innovative therapeutic approaches for rare genetic disorders.
- Lysosomal Storage Disorders
- Pediatric Genetic Conditions
- Enzyme Replacement Therapies
Evaluates novel therapeutic strategies including gene therapy and enzyme replacement approaches.
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