Maria M. B.
- Country
- Poland
- Department
- Klinika Neurologii Rozwojowej
Research Overview
- Phase 3 Study to Evaluate the Efficacy and Safety of ION582 in Children and Adults with Angelman Syndrome
- Phase 3 Randomized, Double‑Blind, Placebo‑Controlled Study of Oral Fenfluramine Hydrochloride in Participants with Rett Syndrome
- Phase 3 Study of Delpacibart Zotadirsen in Duchenne Muscular Dystrophy with Mutations Amenable to Exon 44 Skipping
Data & Insights
Has run more than one trial at 75 of 197 partner sites.
Specializations
Neuromuscular Disorders
The investigator specializes in treating Spinal Muscular Atrophy (SMA) and Duchenne Muscular Dystrophy (DMD), conducting research on innovative therapeutic approaches for these conditions.
- SMA Treatment Optimization
- DMD Therapeutic Development
- Myasthenia Gravis Management
Focuses on evaluating novel treatments including risdiplam, eteplirsen, and emerging therapies for pediatric patients.
Neurological Disorders
Expertise in treating Multiple Sclerosis and Neuromyelitis Optica Spectrum Disorder, with particular attention to pediatric and adolescent populations.
- Relapsing-Remitting MS Treatment
- NMOSD Management
- Neuroimmunological Therapies
Investigates targeted therapies including ocrelizumab, ofatumumab, and inebilizumab.
Epilepsy and Related Conditions
Conducts research in Drug-resistant Epilepsy, Dravet Syndrome, and Tuberous Sclerosis Complex.
- Refractory Seizure Management
- Novel Anticonvulsant Therapies
- Genetic Epilepsy Syndromes
Studies extended-release formulations and investigational compounds for seizure control.
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