Rare diseases
Investigational molecules
Locations
Via Olgettina 58, 20132 Milan, Italy
Clinical research activity includes transfusion-dependent beta-thalassemia, with attention to restoring transfusion independence and improving outcomes in patients with chronic haemoglobin disorders.
The sponsor’s work in this area is centred on gene therapy approaches for inherited anaemias and related blood-cell replacement strategies.
San Raffaele Hospital runs clinical trials in conditions such as Thalassaemia beta, Thalassaemia, Osteopetrosis, Primary immunodeficiency syndrome, among others.
Clinical trials run by this sponsor – those enrolling participants first.
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