Mirjam J.
- Country
- Germany
- Department
- Clinic for Paediatrics and Adolescent Medicine
Research Overview
Data & Insights
Has run more than one trial at 0 of 25 partner sites.
Specializations
Pediatric Oncology and Hematology
Focuses on malignant blood and solid tumors affecting children and adolescents.
- Acute Lymphoblastic Leukemia
- Acute Myeloid Leukemia
- Hodgkin Lymphoma
- Neuroblastoma
- Ewing Sarcoma
- Medulloblastoma
Clinical investigations target disease‑free survival and therapeutic safety in young patients.
Rare Genetic and Metabolic Disorders
Investigates inherited metabolic and lysosomal storage diseases in the pediatric population.
- Pompe disease
- Friedreich’s ataxia
- Niemann‑Pick disease type C
- Mucopolysaccharidosis type II
- Glycogen storage disease type II
- Leber congenital amaurosis
Trials assess disease progression and quality‑of‑life outcomes.
Neurodevelopmental and Neurological Diseases
Addresses seizure disorders, movement disorders, and neurodegenerative conditions in children.
- Focal epilepsy
- Status epilepticus
- Spinal Muscular Atrophy
- Early‑onset Parkinson’s disease
- Ataxia Telangiectasia
- Friedreich’s ataxia
Outcomes include seizure control and motor function preservation.
Immunology and Stem Cell Transplantation
Explores immune‑mediated disorders and hematopoietic stem cell transplantation in pediatric patients.
- Graft‑versus‑host disease
- Hemophilia A
- Hemophilia B
- Immune thrombocytopenia
- Fetal and Neonatal Alloimmune Thrombocytopenia
- Stem cell transplant for acute lymphoblastic leukemia
Research evaluates transplant conditioning and post‑transplant immune reconstitution.
Cardiovascular and Pulmonary Pediatric Conditions
Studies congenital and acquired heart and lung diseases in children.
- Congenital heart malformation
- Pulmonary arterial hypertension
- Idiopathic pulmonary fibrosis
- Hypertrophic cardiomyopathy
- Aortic stenosis
- Acute kidney injury post‑cardiac surgery
Trials aim to improve functional capacity and reduce organ‑specific morbidity.
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