Mireia D. T. R.
- Country
- Spain
- Department
- Pediatric Neurology
Research Overview
- Long-Term Safety, Tolerability, and Efficacy of Iduronate-2-Sulfatase-Fc Polypeptide in Mucopolysaccharidosis Type II Patients from DNLI-E-0002/0007 Studies
- Evaluation of scAAV9.U1a.hSGSH Gene Transfer Therapy with Rebisufligene Etisparvovec in Mucopolysaccharidosis Type IIIA Patients
- Long-term Safety and Tolerability Evaluation of Rebisufligene Etisparvovec in Mucopolysaccharidosis Type IIIA Patients from Gene Therapy Trials
Data & Insights
Has run more than one trial at 11 of 30 partner sites.
Specializations
Lysosomal Storage Disorders
This investigator specializes in treating rare lysosomal storage disorders, with particular focus on Metachromatic Leukodystrophy (MLD) and Niemann-Pick type C disease. Their research extends to various forms of gangliosidoses, including GM1 and GM2 variants.
- Late Infantile MLD Treatment
- GM1/GM2 Gangliosidosis Management
- Niemann-Pick Disease Therapeutics
Their work involves innovative therapeutic approaches for treating these progressive neurological conditions.
Mucopolysaccharidosis Research
The investigator has extensive experience in treating various forms of Mucopolysaccharidosis, particularly focusing on MPS II and MPS IIIA. Their research encompasses both neuronopathic and non-neuronopathic manifestations of these disorders.
- MPS II Treatment Development
- MPS IIIA Gene Therapy
- CNS-targeted Therapeutics
Their research emphasizes long-term safety and efficacy evaluations in pediatric and young adult populations.
Metabolic Disorders
The investigator specializes in treating genetic metabolic conditions, with particular expertise in Ornithine Transcarbamylase (OTC) deficiency. Their research focuses on developing innovative therapeutic approaches for late-onset metabolic disorders.
- OTC Deficiency Management
- Gene Transfer Technologies
- Metabolic Disease Therapeutics
Their work includes investigating novel gene therapy approaches for treating inherited metabolic disorders.
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