Janbernd K.
- Country
- Germany
- Department
- Zentrum für Kinder- und Jugendmedizin, Klinik für Neuropädiatrie und Muskelerkrankungen
Research Overview
- Phase 3 Randomized Double‑Blind Placebo‑Controlled Study of Single Intravenous SGT‑003 Gene Therapy in Ambulant Male Patients with Duchenne Muscular Dystrophy
- Efficacy and Safety Evaluation of Givinostat in Non-Ambulant Duchenne Muscular Dystrophy: A Randomized, Double-Blind, Placebo-Controlled Multicenter Trial
- Efficacy of Calcium Folinate in Kearns-Sayre Syndrome with Cerebral Folate Deficiency: A Randomized Controlled Trial
Data & Insights
Has run more than one trial at 39 of 71 partner sites.
Specializations
Neurological Disorders
This investigator specializes in treating pediatric migraine conditions, with particular emphasis on developing therapeutic approaches for children aged 6 to 17 years.
- Pediatric Migraine Management
- Acute Migraine Treatment
- Long-term Pain Management
Their research extends to evaluating innovative treatment options for young patients experiencing migraine episodes.
Rare Diseases
The investigator conducts research in rare genetic disorders, particularly focusing on Kearns-Sayre Syndrome (KSS) and associated neurological conditions.
- Mitochondrial Disorders
- Cerebral Folate Deficiency
- Metabolic Therapy
Their work includes investigating therapeutic interventions for rare neurological conditions using folinic acid supplementation.
Therapeutic Development
The investigator explores novel therapeutic approaches for neurological conditions, with a focus on safety and efficacy evaluations in pediatric populations.
- Drug Safety Assessment
- Pediatric Clinical Research
- Treatment Optimization
Their research encompasses both acute and long-term therapeutic interventions for neurological disorders.
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