Congenital thrombotic thrombocytopenic purpura
Clinical research in cTTP focuses on ADAMTS13 replacement and the management of microvascular thrombosis linked to severe deficiency of this enzyme. The studies address both prophylactic treatment and on-demand therapy in patients with inherited thrombotic disease.
- ADAMTS13 replacement therapy
- Thrombotic microangiopathy
- Bleeding and clotting balance
The sponsor’s work in this area is centered on haematology and inherited disorders of coagulation control.
Hereditary severe von Willebrand disease in children
Research in severe von Willebrand disease addresses control of nonsurgical bleeding and support for perioperative haemostasis in paediatric patients. The therapeutic interest includes recombinant von Willebrand factor and its use in children with inherited bleeding disorders.
- Paediatric haemostasis
- Bleeding episode control
- Inherited von Willebrand disease
This area reflects a focus on paediatric haematology and the treatment of rare coagulation disorders.
Primary immunodeficiency diseases
Clinical activity in primary immunodeficiency centers on long-term support for patients with impaired immune function, with attention to immunoglobulin replacement and ongoing treatment tolerability. The research landscape includes chronic care for inherited immune defects and related infection risk.
- Immunoglobulin therapy
- Immune system deficiency
- Long-term treatment tolerability
The sponsor is active in clinical immunology and disorders marked by recurrent infections and immune dysfunction.
