Respiratory Tract Diseases

The department is conducting studies on severe eosinophilic asthma in children, testing new biologic agents to lower attack frequency.

  • Asthma
  • Severe uncontrolled asthma
  • Pediatric severe asthma

Ongoing trials evaluate benralizumab and tezepelumab for safety and efficacy in reducing exacerbations and improving lung function.

Immune System Diseases

Research focuses on rare autoinflammatory diseases and broader autoimmune conditions, seeking targeted therapies that modulate immune pathways.

  • Autoinflammatory diseases (NLRC4‑GOF, XIAP deficiency, CDC42 mutation)
  • Immunoglobulin G4‑related disease
  • Generalized Myasthenia Gravis
  • Systemic Lupus Erythematosus

Trials assess agents such as MAS825, inebilizumab, and anifrolumab for disease control and safety in pediatric populations.

Digestive System Diseases

The site is evaluating novel biologics for chronic inflammatory bowel conditions, aiming to achieve durable remission in young patients.

  • Crohn’s disease
  • Ulcerative colitis
  • Inflammatory Bowel Disease

Studies test mirikizumab, guselkumab, and long‑term vedolizumab therapy to improve mucosal healing and quality of life.

Hemic and Lymphatic Diseases

Investigations target bleeding disorders and pediatric blood cancers, exploring both replacement strategies and immune‑based treatments.

  • Hemophilia A and B
  • Philadelphia chromosome‑positive B‑ALL
  • Acute Myeloid Leukemia with NPM1 mutation
  • Hematopoietic stem cell transplantation in ALL

Trials include marstacimab for hemophilia, blinatumomab as post‑transplant maintenance, and protocols to refine transplant conditioning.

Musculoskeletal and Neurological Diseases

Pediatric rare‑disease programs evaluate disease‑modifying agents for muscle and nerve disorders, aiming to preserve function and delay progression.

  • Duchenne muscular dystrophy
  • CACNA1A‑related ataxia disorders
  • Metachromatic Leukodystrophy
  • Developmental and Epileptic Encephalopathies

Ongoing studies test givinostat, N‑Acetyl‑L‑Leucine, and gene‑replacement therapy HGT‑1110 for safety and functional improvement.

Hormonal and Congenital Disorders

The hospital investigates treatments for inherited and hormonal conditions that affect growth, kidney function, and metabolic balance.

  • Congenital adrenal hyperplasia
  • Alport syndrome
  • Chronic kidney disease

Clinical work includes evaluating atumelnant for adrenal disease control and BAY 3401016 to reduce albuminuria in Alport syndrome patients.