Mirian J.
- Country
- The Netherlands
- Department
- Metabolic Department
Research Overview
- Phase 2a Study on Safety, Tolerability, and Pharmacodynamics of OMT-28 in Patients with Primary Mitochondrial Disease-Associated Myopathy/Cardiomyopathy
- Efficacy and Safety of Oral TTI-0102 in Mitochondrial Encephalomyopathy, Lactic Acidosis, and Stroke-like Episodes: A Randomized, Double-Blind, Placebo-Controlled Trial
- A Phase III, randomised, double-blind, placebo-controlled, parallel-group, pivotal trial to assess the efficacy and safety of sonlicromanol in adult subjects with a genetically confirmed mitochondrial DNA tRNALeu(UUR) m.3243A>G variant.
Data & Insights
Has run more than one trial at 2 of 14 partner sites.
Specializations
Mitochondrial Disorders
The investigator specializes in treating rare mitochondrial diseases, with particular emphasis on Primary Mitochondrial Disease (PMD) and MELAS syndrome.
- PMD with myopathy and cardiomyopathy manifestations
- Inflammatory conditions in mitochondrial disorders
- MELAS treatment approaches
Actively involved in evaluating therapeutic interventions for patients with mitochondrial-related complications.
Therapeutic Focus
The investigator concentrates on developing treatments for metabolic disorders with particular attention to novel therapeutic compounds.
- Innovative oral therapeutics
- Long-term treatment strategies
- Pharmacodynamic assessments
Evaluates novel compounds targeting specific mitochondrial disease manifestations.
Clinical Expertise
Focuses on treating complex neurometabolic conditions with emphasis on stroke-like episodes and metabolic complications.
- Biomarker analysis
- Safety and tolerability assessments
- Treatment response evaluation
Specializes in managing patients with complex mitochondrial disease presentations.
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