Congenital, Hereditary, and Neonatal Diseases and Abnormalities
At Hospital Femme Mere Enfant, researchers address rare metabolic conditions, notably Ornithine transcarbamylase deficiency. Studies are designed to monitor long‑term safety of gene‑delivery therapies in adults with late‑onset disease.
- Gene therapy safety monitoring
- Long‑term metabolic outcome assessment
- Adult participants with late‑onset presentation
The department involves 17 investigators and contributes to the broader effort in congenital disease research across Europe.
Diseases – Female Urogenital Diseases and Pregnancy Complications
Clinical work in the female urogenital field targets advanced ovarian, tubal and peritoneal cancers. The aim is to test combined chemotherapy and targeted maintenance regimens to extend progression‑free survival after optimal surgery.
- Carboplatin‑paclitaxel backbone with targeted agents
- Maintenance with niraparib ± bevacizumab
- Progression‑free survival after cytoreductive surgery
These investigations reflect the site’s commitment to improving outcomes for women with high‑risk gynecologic malignancies.
Diseases – Immune System Diseases
The immune system disease program evaluates novel oral and injectable agents for conditions such as Behçet’s disease and Crohn’s disease. Trials seek to reduce inflammatory activity and improve quality of life, especially in pediatric patients.
- Apremilast for oral ulcer management
- Risankizumab efficacy in pediatric Crohn’s disease
- Safety profiling of immune‑modulating drugs
By focusing on immune‑mediated disorders, the team supports the development of therapies that address both systemic and mucosal symptoms.
Diseases – Neoplasms
Within the neoplasm portfolio, the hospital examines cutting‑edge strategies for high‑grade ovarian and related cancers. Research centers on integrating anti‑angiogenic drugs with standard chemotherapy and subsequent maintenance therapy.
- Anti‑angiogenic bevacizumab integration
- Post‑surgical maintenance strategies
- Evaluation of disease‑free survival metrics
The goal is to achieve longer disease‑free intervals while maintaining tolerability for patients after extensive surgical treatment.
Phenomena and Processes – Digestive System and Oral Physiological Phenomena
Studies of digestive system phenomena address gastrointestinal inflammation and oral health, including Crohn’s disease and oral ulcers linked to Behçet’s. The purpose is to assess biologic efficacy and symptom relief in children and adolescents.
- Biologic pharmacokinetics in children
- Mucosal healing endpoints
- Oral ulcer symptom control
These efforts aim to provide age‑appropriate therapeutic options for disorders that affect both the gut and oral cavity.
Phenomena and Processes – Immune system processes
The immune system processes research focuses on pediatric Systemic Lupus Erythematosus. Trials are designed to identify optimal dosing and confirm safety while measuring disease activity improvements.
- Dose‑finding for anifrolumab in youth
- BICLA response as efficacy marker
- Safety surveillance in severe SLE
Such work contributes to expanding treatment possibilities for young patients living with severe autoimmune disease.
