Hopital Necker Enfants Malades

The Service de Gastro-entérologie et Nutrition Pédiatriques at Hôpital Necker Enfants Malades focuses on improving care for children with gastrointestinal disorders and nutritional challenges. Clinical research aims to evaluate new therapies, refine diagnostic tools, and enhance long‑term health outcomes for young patients.

• Investigating novel treatments for Crohn’s Disease and Ulcerative Colitis in pediatric populations
• Assessing nutritional interventions for intestinal malabsorption in preterm infants
• Exploring therapies to reduce proteinuria and preserve renal function in children with kidney‑related digestive complications
• Evaluating safety and efficacy of biologics such as mirikizumab and ixekizumab for inflammatory bowel disease
• Studying the impact of gut‑microbiome modulation on disease activity and growth parameters

These studies aim to bring effective, child‑friendly treatments to the bedside and support better growth, development, and quality of life for affected children.

Research at the site addresses rare genetic conditions that affect skeletal development and metabolism. Trials are designed to test innovative gene‑based and peptide therapies that could alter disease trajectories and improve physical outcomes.

• Long‑term safety and growth benefits of TransCon CNP in children with achondroplasia
• Evaluating the efficacy of BMN 111 for height gain in achondroplastic patients
• Assessing infigratinib and other agents for bone growth in pediatric achondroplasia
• Investigating metabolic impacts of therapies for congenital adrenal hyperplasia
• Studying treatment approaches for rare vascular malformations affecting facial and oral structures

By targeting the underlying molecular pathways, these trials seek to provide lasting improvements in stature, endocrine balance, and overall health for children with congenital growth disorders.

The department conducts extensive studies on pediatric blood and immune diseases, ranging from hemophilia to immune deficiencies. The primary goal is to introduce safer, more effective therapies and to reduce disease‑related complications.

• Evaluating gene‑therapy approaches for severe hemophilia A using AAV vectors
• Testing novel immunoglobulin‑targeting agents in children with autoimmune hemolytic anemia
• Assessing the safety of long‑acting antibodies for HIV remission in early infection
• Studying treatments for EBV‑related post‑transplant lymphoproliferative disease in kidney transplant recipients
• Investigating the efficacy of ravulizumab for transplant‑associated thrombotic microangiopathy

These investigations aim to enhance disease control, lower bleeding risk, and improve transplant outcomes for young patients.

Pediatric neuro‑oncology and neurodevelopmental conditions are a key focus, with trials exploring targeted therapies and supportive care strategies to improve neurological function and survival.

• Assessing the efficacy of ONC201 in children with diffuse intrinsic pontine glioma and related midline gliomas
• Evaluating radiprodil for seizure reduction in GRIN‑related neurodevelopmental disorder
• Testing early rituximab infusion to prevent EBV infection in pediatric kidney transplant recipients
• Studying gene‑editing therapies for spinal muscular atrophy
• Exploring treatments for type 1 interferonopathies and related autoimmune encephalopathies

The research strives to provide disease‑modifying options that can preserve neurological development and extend survival for children with severe brain disorders.

A broad spectrum of metabolic and rare genetic diseases are under investigation, with an emphasis on innovative drug candidates and precision medicine approaches.

• Testing povetacicept to reduce proteinuria in immunoglobulin A nephropathy
• Evaluating stiripentol for lowering urinary oxalate in primary hyperoxaluria
• Assessing mavorixafor to improve neutrophil counts in chronic neutropenia
• Investigating crinecerfont for adrenal steroid regulation in congenital adrenal hyperplasia
• Studying therapies for metabolic bone disease in children with PIK3CA‑related overgrowth spectrum

These studies aim to deliver targeted treatments that address the root causes of metabolic dysfunctions, offering hope for improved quality of life.