Dariusz R.
- Country
- Poland
- Department
- Klinika Pediatrii, Pawlinski@su.krakow.pl
Research Overview
- Efficacy and Safety of Pegtibatinase Added to Standard of Care in Participants with Classical Homocystinuria Due to Cystathionine Beta Synthase Deficiency
- Evaluation of Hydroxypropylbetadex and Standard of Care Versus Placebo in Niemann-Pick Disease Type C1: A Phase 3 Randomized, Double-Blind, Multicenter Study
- Evaluation of Triheptanoin Versus Even-Chain Medium-Chain Triglycerides on Major Clinical Events in Pediatric Long-Chain Fatty Acid Oxidation Disorders
Data & Insights
Has run more than one trial at 6 of 28 partner sites.
Specializations
Metabolic Disorders
The investigator specializes in treating rare metabolic conditions, with particular emphasis on glycogen storage disorders, specifically focusing on glycogenosis type Ib and its associated neutropenia complications.
- Glycogen Storage Disease Management
- Neutropenia Treatment
- Metabolic Disease Therapeutics
Their research extends to innovative therapeutic approaches using novel compounds for managing rare metabolic conditions.
Lysosomal Storage Disorders
The investigator focuses on treating Niemann-Pick Disease Type C1, a rare progressive genetic disorder affecting lipid metabolism and transport. Their expertise includes evaluating novel therapeutic approaches for managing this complex condition.
- Neurological Manifestations
- Disease Progression Assessment
- Therapeutic Interventions
Their work encompasses comprehensive disease severity assessment and management strategies.
Fatty Acid Metabolism
The investigator specializes in treating Long-chain Fatty Acid Oxidation Disorders, focusing on pediatric patients with metabolic dysfunction affecting fat metabolism and energy production.
- Energy Metabolism Disorders
- Nutritional Management
- Clinical Event Prevention
Their research includes comparative studies of different therapeutic approaches for managing fatty acid metabolism disorders.
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