Fanny M.
- Country
- France
- Department
- Service de Génétique Médicale
Research Overview
- Efficacy and Safety Evaluation of Leriglitazone in Adult Males with Cerebral Adrenoleukodystrophy: A Randomized, Placebo-Controlled Clinical Trial
- A Phase 2 Safety, Tolerability, and Proof-of-Concept Study of VGL101 in Patients With Adult-Onset Leukoencephalopathy With Axonal Spheroids and Pigmented Glia (ALSP)
- 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 10 partner sites.
Specializations
Neurodegenerative Disorders
This investigator specializes in rare neurological conditions, with particular emphasis on cerebral adrenoleukodystrophy (cALD) and adult-onset leukoencephalopathy.
- White Matter Disorders
- Genetic Neurological Conditions
- Progressive Brain Disorders
The research focus includes therapeutic interventions for rare genetic disorders affecting brain white matter and neurological function.
Therapeutic Development
The investigator studies novel therapeutic approaches for rare neurological diseases, including leriglitazone and iluzanebart as potential treatment options.
- Innovative Treatment Approaches
- Disease-Modifying Therapies
- Therapeutic Safety Assessment
Areas of interest encompass the development and evaluation of treatments targeting specific neurological pathways.
Clinical Outcomes
The investigator focuses on evaluating treatment outcomes in rare neurological disorders, with particular attention to survival rates and disease progression.
- Treatment Efficacy Analysis
- Patient Safety Monitoring
- Disease Management Strategies
Research activities center on assessing therapeutic interventions' impact on patient outcomes and quality of life.
Related Investigators
Researchers in similar fields or departments
Want to know more about this investigator?
We can share extended profile information and contact details.
Is something out of date?
Tell us what needs correcting and we'll update this profile.