Congenital, Hereditary, and Neonatal Diseases and Abnormalities

The centre is investigating innovative treatments for classic congenital adrenal hyperplasia. Research includes evaluating hormone‑modulating agents to reduce glucocorticoid dependence.

  • Classic Congenital Adrenal Hyperplasia (CAH)
  • Congenital adrenal hyperplasia
  • Lu AG13909 study in CAH patients

These trials aim to improve disease control and quality of life by testing new endocrine therapies.

Digestive System Diseases

Researchers are assessing subcutaneous biologics for patients with ulcerative colitis. Parallel studies examine efficacy in pediatric subjects with moderate to severe Crohn’s disease.

  • Moderately to severely active ulcerative colitis
  • Moderately to severely active Crohn’s disease
  • Pharmacokinetic profiling of vedolizumab in children

The purpose is to expand therapeutic options and improve long‑term remission rates in inflammatory bowel disease.

Hormonal Diseases

The site evaluates novel inhibitors that target adrenal steroid synthesis for congenital adrenal hyperplasia. The goal is to lower daily glucocorticoid dosage while maintaining hormonal balance.

  • Crinecerfont efficacy in reducing glucocorticoid use
  • Adrenal androgen control assessments
  • Alternative dosing regimens for steroid‑sparing therapy

These studies focus on safer hormone management and reduced side‑effects for patients with endocrine disorders.

Immune System Diseases

Clinical investigations target autoimmune conditions such as rheumatoid arthritis and systemic lupus erythematosus. Trials test disease‑modifying agents and biologics to curb inflammation.

  • Early rheumatoid arthritis with methotrexate combination
  • Systemic lupus erythematosus and cutaneous lupus erythematosus
  • Enpatoran dose‑response in skin manifestations of lupus

The overarching aim is to validate effective therapies that can halt disease progression and preserve joint and organ function.

Respiratory Tract Diseases

Studies are underway to improve management of chronic airway conditions, including chronic obstructive pulmonary disease. Additional work examines allergic rhinitis and asthma comorbidities.

  • Inhaled triple therapy (budesonide/glycopyrronium/formoterol) in uncontrolled asthma
  • Birch and grass pollen allergen immunotherapy for seasonal allergic rhinitis
  • Respiratory tract infection prevention in children using sublingual tablets

These trials seek to reduce exacerbations, enhance lung function, and lower infection rates through new pharmacologic approaches.

Skin and Connective Tissue Diseases

The centre conducts extensive research on inflammatory skin disorders such as atopic dermatitis. Multiple biologics and small molecules are evaluated for efficacy and safety.

  • Upadacitinib versus dupilumab in pediatric moderate‑to‑severe atopic dermatitis
  • Barzolvolimab trials in chronic spontaneous urticaria and cold‑induced urticaria
  • Galvokimig dose‑response in moderate‑to‑severe atopic dermatitis
  • Rezpegaldesleukin in biologic‑naïve atopic dermatitis patients
  • Psoriasis plaque treatment with SCD‑044
  • Hidradenitis suppurativa evaluation with lutikizumab
  • Prurigo nodularis and nummular eczema studies

The objective is to bring forward new therapeutic options that achieve rapid skin clearance and improve patients’ quality of life.

Immune System Phenomena

Research explores underlying immune mechanisms that drive allergic and autoimmune responses, focusing on eosinophilic inflammation. Trials assess targeted agents that modulate specific immune pathways.

  • Dexpramipexole impact on severe eosinophilic asthma exacerbations
  • Leukotriene pathway inhibition in chronic spontaneous urticaria
  • Biomarker analysis for treatment response in atopic dermatitis

These investigations aim to deepen understanding of immune dysregulation and identify precise interventions for related diseases.