4 results
We propose a phase II study (intervention) in patients with severe generalized recessive dystrophic EB receiving reduced toxicity conditioning chemotherapy followe by cord blood transplantation with co-infusion of mesenchymal stromal cell units.…
1.3. Study Design Rationale The ATLAS-PPX trial (ALN-AT3SC-009) is a multicenter, multinational, open-label Phase 3 switching study designed to demonstrate the efficacy and safety of fitusiran in patients with hemophilia A or B, with inhibitory…
Primary Objective: Validation of T1rho&T2mapping in healthy volunteers and patients with an OCD of the talusSecondary objective: To quantify T1rho & T2 mapping in patients with osteochondrial defects against healthy volunteers with…
The ATLAS-A/B trial (ALN-AT3SC-004) is a multicenter, multinational, randomized, openlabel Phase 3 study designed to demonstrate the efficacy and safety of fitusiran in patients with hemophilia A or B without inhibitory antibodies to FVIII or FIX…