Magalie B.
- Country
- France
- Department
- Service de neuropédiatrie
Research Overview
- Long-Term Safety and Efficacy of Intrathecal Cebsulfase Alfa in Pediatric Patients with Late Metachromatic Leukodystrophy: An Extension of Study HGT-MLD-070
- Efficacy and Safety of Oral TTI-0102 in Mitochondrial Encephalomyopathy, Lactic Acidosis, and Stroke-like Episodes: A Randomized, Double-Blind, Placebo-Controlled Trial
Data & Insights
Has run more than one trial at 1 of 14 partner sites.
Specializations
Neurometabolic Disorders
This investigator specializes in treating rare genetic disorders affecting the nervous system, with particular emphasis on mitochondrial diseases and leukodystrophies.
- MELAS Syndrome Management
- Metachromatic Leukodystrophy Treatment
The investigator focuses on evaluating novel therapeutic approaches for patients with these challenging neurometabolic conditions.
Genetic Medicine
Expertise in treating inherited metabolic disorders, particularly focusing on enzyme replacement therapies and innovative treatment modalities for rare genetic conditions.
- Metabolic Disease Management
- Genetic Disorder Therapeutics
The investigator works with advanced therapeutic interventions for patients with inherited metabolic disorders.
Pediatric Neurology
Specialized focus on treating neurological conditions in children, particularly progressive neurometabolic disorders requiring long-term therapeutic intervention.
- Pediatric Metabolic Disorders
- Neurological Disease Management
The investigator's work encompasses comprehensive care approaches for young patients with complex neurological conditions.
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