Rare diseases
Investigational molecules
Locations
A Study of Gene Therapy Using Modified Stem Cells for Patients with Transfusion-Dependent Beta-Thalassemia Who Need Regular Blood Transfusions
ItalyStudy on the Safety and Effectiveness of CTX001 for Children with Transfusion-Dependent Beta-Thalassemia Using Exagamglogene Autotemcel and a Drug Combination
GermanyLong-term safety study of oral etavopivat tablets in children, adolescents and adults with sickle cell disease or thalassaemia who completed previous etavopivat treatment
FranceStudy on Long-Term Safety of Luspatercept for Patients with Myelodysplastic Syndrome, Beta-Thalassemia, or Myelofibrosis Who Previously Participated in Luspatercept Trials
BulgariaLong-term Safety Study of Exagamglogene Autotemcel for Patients with Sickle Cell Disease or Transfusion-Dependent Thalassemia
BelgiumStudy on Luspatercept for Improving Brain Oxygenation in Patients with Non-Transfusion Dependent Thalassemia
The NetherlandsPhase 3 study of mitapivat for effectiveness and safety in children with non‑transfusion‑dependent alpha or beta thalassemia
GreecePhase 3 Study of Mitapivat to Reduce Transfusions in Children with Alpha or Beta Transfusion‑Dependent Thalassemia
GreeceStudy on the Effectiveness and Safety of Mitapivat for Patients with Non-Transfusion-Dependent Alpha or Beta Thalassemia
BulgariaStudy on the Effects of Mitapivat for Patients with Transfusion-Dependent Alpha or Beta Thalassemia
BulgariaStudy on Long-Term Safety of Ruxolitinib, Panobinostat, and Siremadlin for Patients Continuing Treatment from Previous Studies
GermanyTell us about your condition – we search every trial in Europe and connect you with the right site.
