Małgorzata W. C.
- Country
- Poland
- Department
- Diabetology
Research Overview
- A 52-week randomized, double-blind, placebo-controlled, multi-center Phase 2b study with a 52-week blinded extension and an optional open-label extension—assessing the safety and efficacy of frexalimab, a CD40L-antagonist monoclonal antibody, for the preservation of pancreatic β-cell function in adults and adolescents with newly diagnosed type 1 diabetes on insulin therapy
- Evaluation of Sotatercept (MK-7962) Safety, Tolerability, Pharmacokinetics, and Pharmacodynamics in Pediatric Pulmonary Arterial Hypertension
- Long-Term Safety Evaluation of Teplizumab in Pediatric and Adolescent Patients with Recent-Onset Type 1 Diabetes Mellitus: An Extension of Study PRV-031-001
Data & Insights
Has run more than one trial at 20 of 98 partner sites.
Specializations
Inflammatory Bowel Disease and Gastroenterology
Clinical research activity is centered on pediatric and adolescent ulcerative colitis, Crohn's disease, and related inflammatory bowel diseases, with interest in treatment-resistant intestinal inflammation, mucosal healing, and long-term disease control.
- Ulcerative colitis and Crohn's disease
- Chronic pouchitis and complex perianal fistulas
- Biologic and targeted immunotherapy
Therapeutic involvement also extends to pouchitis, perianal fistulizing disease, and maintenance strategies for persistent intestinal inflammation in younger patients.
Hepatology and Rare Cholestatic Disorders
The investigator is involved in studies of pediatric biliary atresia, cholestatic pruritus, and other hepatobiliary diseases affecting liver function and bile flow.
- Biliary atresia and post-surgical liver outcomes
- Cholestatic pruritus
- Rare liver disease management
Additional interest includes Wilson’s disease and therapeutic approaches aimed at preserving liver health in children with complex hepatobiliary conditions.
Neuroimmunology and Epilepsy
Research activity includes pediatric multiple sclerosis, neuromyelitis optica spectrum disorder, MOGAD, and ataxia-telangiectasia, alongside seizure-related disorders and neurodevelopmental syndromes.
- Relapsing multiple sclerosis
- Neuromyelitis optica spectrum disorder and MOGAD
- Tuberous sclerosis complex and focal cortical dysplasia
Therapeutic focus also covers epilepsy, refractory seizures, and GRIN-related neurodevelopmental disorder, reflecting interest in neurologic disease modification and symptom control.
Pediatric Metabolic, Genetic, and Renal Disorders
The investigator is active in rare inherited conditions including glycogen storage disease type 1a, autosomal recessive polycystic kidney disease, Fabry disease, Niemann-Pick disease type C1, and primary immunodeficiency diseases.
- Chronic kidney disease and proteinuria
- Metabolic liver and storage disorders
- Inherited immune and renal disease
Other areas include long-chain fatty acid oxidation disorders, spinal muscular atrophy, and growth hormone deficiency, with attention to pediatric rare-disease therapeutics.
Endocrine, Cardiopulmonary, and Oncology-Related Pediatric Care
Clinical interests also span type 1 diabetes mellitus, obesity, pulmonary arterial hypertension, and selected pediatric oncology conditions such as hepatoblastoma, lymphoblastic lymphoma, and medulloblastoma.
- Type 1 diabetes and endocrine regulation
- Pulmonary arterial hypertension
- Pediatric solid and hematologic malignancies
Additional therapeutic interest includes gastroesophageal reflux disease, myopia, and neurogenic detrusor overactivity in pediatric populations.
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