Liesbeth D. W.
- Country
- Belgium
- Department
- Paediatric neurology
Research Overview
- Phase 3 Study of Delpacibart Zotadirsen in Duchenne Muscular Dystrophy with Mutations Amenable to Exon 44 Skipping
- Phase 3 Randomized Double‑Blind Placebo‑Controlled Study of Single Intravenous SGT‑003 Gene Therapy in Ambulant Male Patients with Duchenne Muscular Dystrophy
- Phase 2 Open‑Label Extension of ENTR‑601‑45 (with ENTR‑601‑44) in Exon‑Skipping‑Amenable Duchenne Muscular Dystrophy: Safety, Tolerability, PK and Efficacy
Data & Insights
Has run more than one trial at 41 of 73 partner sites.
Specializations
Neuromuscular Disorders
The investigator specializes in treating progressive muscle disorders, with particular emphasis on Duchenne Muscular Dystrophy (DMD) and Spinal Muscular Atrophy (SMA). Their clinical focus encompasses genetic-based therapeutic approaches for these conditions.
- DMD Treatment Innovations
- SMA Management Strategies
- Gene-targeted Therapies
Their work involves evaluating novel therapeutic compounds for treating hereditary muscle disorders.
Genetic Medicine
The investigator focuses on exon skipping therapies and gene modulation approaches for treating neuromuscular conditions. Their expertise includes evaluation of various genetic treatment modalities targeting specific mutations.
- Mutation-specific Treatments
- Gene Expression Modification
- Personalized Genetic Interventions
Their research encompasses long-term safety and efficacy assessment of genetic therapeutics.
Clinical Assessment
The investigator specializes in evaluating motor function and disease progression in neuromuscular disorders. Their work includes comprehensive assessment of patient mobility and muscle strength.
- Functional Mobility Testing
- Long-term Safety Monitoring
- Treatment Response Assessment
They utilize specialized assessment tools to measure therapeutic outcomes in muscle disorders.
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