IRCCS Istituto Giannina Gaslini

The IRCCS Istituto Giannina Gaslini in Genoa is actively studying juvenile idiopathic arthritis, systemic juvenile idiopathic arthritis, lupus nephritis, Behçet’s disease, and other inflammatory conditions. This research focuses on improving symptom control, guiding treatment decisions, and finding safer long-term ways to manage chronic immune-related disease in children and adolescents.

• Juvenile idiopathic arthritis and treatment withdrawal strategies
• Lupus nephritis and related autoimmune kidney disease
• Behçet’s disease, Still’s disease, and other systemic inflammatory disorders

These studies aim to refine care by testing whether personalized approaches can better balance disease control and treatment burden.

Major research at this site centers on pediatric cancers, especially acute lymphoblastic leukemia, lymphoblastic lymphoma, neuroblastoma, medulloblastoma, glioma, and acute myeloid leukemia. The studies explore new cancer treatments, molecularly targeted therapies, and better strategies for children whose disease has returned or not responded to standard care.

• Relapsed or refractory leukemia and lymphoma
• High-risk neuroblastoma and other solid tumors
• Brain tumors such as medulloblastoma, ependymoma, and diffuse intrinsic pontine glioma

The overall goal is to improve survival, reduce relapse, and match treatments more closely to each child’s tumor biology.

Clinical trials at IRCCS Istituto Giannina Gaslini also address a broad range of kidney diseases, including chronic kidney disease, proteinuria, nephrotic syndrome, IgA nephropathy, Alport syndrome, and post-transplant complications. This work is designed to improve kidney protection, reduce protein loss in urine, and support better outcomes after transplant.

• Chronic kidney disease with proteinuria in children
• Nephrotic syndrome and other glomerular diseases
• Adolescent renal transplant care and immunosuppression management

Research in this area is largely aimed at evaluating treatments that may preserve kidney function and improve long-term disease control.

The site has strong activity in neurological disorders such as Dravet syndrome, Lennox-Gastaut syndrome, developmental and epileptic encephalopathies, spinal muscular atrophy, Duchenne muscular dystrophy, and Rett syndrome. These trials are focused on testing new therapies, improving seizure control, and understanding how treatments affect function and daily life.

• Epilepsy and severe seizure syndromes
• Neuromuscular diseases such as spinal muscular atrophy and Duchenne muscular dystrophy
• Neurodevelopmental disorders including Rett syndrome and CDKL5 deficiency disorder

These studies aim to improve both symptom management and longer-term neurological outcomes in children.

Respiratory research at the site includes cystic fibrosis, severe asthma, bronchiectasis, and chronic lung disease. The trials are examining new therapies and better ways to maintain lung function, reduce flare-ups, and support children with chronic breathing problems.

• Cystic fibrosis and long-term respiratory care
• Severe uncontrolled asthma in children
• Chronic lung disease in premature infants and other pediatric patients

This work is aimed at improving everyday respiratory health and lowering the burden of long-term lung disease.

Within the U.O.C. Clinica Pediatrica-Endocrinologia, the research portfolio includes growth hormone deficiency, Prader-Willi syndrome, classic congenital adrenal hyperplasia, Glycogen Storage Disease Type Ia, Wilson disease, and other metabolic disorders. These studies are designed to evaluate new treatments, improve disease control, and address complications that affect growth, appetite, and metabolism.

• Growth and endocrine disorders in children
• Prader-Willi syndrome and appetite-related symptoms
• Inherited metabolic diseases including Wilson disease and glycogen storage disorders

The research aims to support more effective, child-friendly approaches for long-term endocrine and metabolic care.

Clinical trials here also cover heart failure, hemophilia, sickle cell disease, thrombocytopenia, and other hematologic disorders. The focus is on improving disease stability, preventing bleeding or vaso-occlusive events, and testing treatments that may offer better control of chronic blood-related conditions.

• Hemophilia and bleeding disorders
• Sickle cell disease and crisis prevention
• Heart failure and related pediatric cardiovascular care

These studies are intended to strengthen supportive care and evaluate therapies that may improve safety and quality of life.

Research at the site includes immune system diseases such as chronic graft-versus-host disease, hemophagocytic syndromes, post-transplant viral reactivation, and immune-mediated inflammatory disorders. The studies seek to improve treatment effectiveness, reduce complications after transplant, and better control severe immune activation.

• Post-transplant complications after hematopoietic stem cell transplant
• Chronic inflammatory immune disorders
• Viral reactivation and transplant-related immune dysfunction

This line of research reflects a strong interest in safer, more targeted management of complex pediatric immune conditions.

The site also contributes to studies in diagnostic techniques, therapeutics, and surgical procedures, including research on anesthesia and outcomes around planned operations in young children. These trials are aimed at improving safety, refining care pathways, and supporting better recovery after procedures.

• Anesthesia and perioperative care in young children
• Diagnostic and therapeutic techniques for pediatric care
• Surgical outcomes and recovery support

Work in this area complements the broader pediatric research program by improving how care is delivered across different clinical settings.