3 results
This study will evaluate the safety, tolerability, and pharmacokinetics of single and multiple ascending doses of GDC-0134 in patients with amyotrophic lateral sclerosis (ALS).The long term safety and tolerability of up to 48 weeks of GDC-0134 will…
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…
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…