Sabrina S.
- Country
- France
- Department
- Service de Neurologie - Système Nerveux Périphérique et Muscle
Research Overview
- Efficacy of Rituximab Versus Placebo in Anti-MAG Neuropathy Patients with Predicted Good Response: A Randomized Controlled Trial
- Open-label extension study of KL1333 for long-term safety, tolerability, and efficacy in adults with primary mitochondrial disease
- A Phase 2 Randomized, Double-blind, Placebo-controlled Study to Evaluate the Effect of EDG-5506 on Safety, Biomarkers, Pharmacokinetics, and Functional Measures in Adults and Adolescents with Becker Muscular Dystrophy
Data & Insights
Has run more than one trial at 147 of 290 partner sites.
Specializations
Neuromuscular Disorders
This investigator specializes in treating various neuromuscular disorders, with particular emphasis on muscular dystrophies including Becker Muscular Dystrophy, Facioscapulohumeral Muscular Dystrophy, and Myotonic Dystrophy Type 1.
- Becker Muscular Dystrophy (BMD)
- Facioscapulohumeral Muscular Dystrophy (FSHD)
- Myotonic Dystrophy Type 1 (DM1)
Autoimmune Conditions
The investigator focuses on autoimmune neurological disorders, particularly Myasthenia Gravis and inflammatory neuropathies. Their work includes research on chronic inflammatory demyelinating polyneuropathy and dermatomyositis.
- Generalized Myasthenia Gravis
- Chronic Inflammatory Demyelinating Polyneuropathy
- Dermatomyositis
- Anti-MAG Neuropathy
Metabolic and Genetic Disorders
The investigator studies rare genetic and metabolic disorders affecting the neuromuscular system, including mitochondrial diseases and glycogen storage disorders.
- Primary Mitochondrial Disease
- Glycogen Storage Disease Type II
- Charcot-Marie-Tooth Disease
- Inclusion Body Myositis
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