Rare diseases
Investigational molecules
Locations
Via Varese 16 B, 00185 Rome, Italy
Research activity in pediatric hereditary bone disease includes autologous hematopoietic stem and progenitor cell approaches for osteopetrosis linked to TCIRG1 mutations, with attention to safety and clinical efficacy.
The sponsor’s work in this area centers on genetic correction strategies for severe inherited skeletal disease affecting children.
Fondazione Telethon Ets runs clinical trials in conditions such as Thalassaemia beta, Thalassaemia, Osteopetrosis, Wiskott-Aldrich syndrome, among others.
Clinical trials run by this sponsor – those enrolling participants first.
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